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GBT’s Oxbryta™ (voxelotor) tablets is approved for the treatment of sickle cell disease

Oxbryta™ (voxelotor) tablets Now Approved

On behalf of GBT, we are happy to share that Oxbryta (pronounced ox-brye-ta) is now approved by the U.S. Food and Drug Administration (FDA). Oxbryta is a prescription medicine used for the treatment of sickle cell disease in adults and children 12 years of age and older.1 It is not known if Oxbryta is safe and effective in children below 12 years of age.1
This indication is approved under accelerated approval based on increase in hemoglobin (Hb). Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trial(s).2
This approval has been the result of years of collaboration with the community, and we are so grateful for your role in helping GBT get this to patients quickly!
1) Oxbryta is the brand name for voxelotor. This approval represents a historic one, as Oxbryta is the first sickle cell disease drug approved under both the Breakthrough Therapy and Accelerated Approval designations by the FDA.
· Voxelotor is a hemoglobin S (HbS) polymerization inhibitor.2
· Nonclinical studies suggest that voxelotor may inhibit red blood cell (RBC) sickling, improve RBC deformability, and reduce whole blood viscosity.2
· It is a prescription medicine (tablet) taken by mouth once daily, every day.1
2) Oxbryta should not be taken if a patient has had an allergic reaction to voxelotor or any of the ingredients in Oxbryta. Oxbryta can cause side effects including: headache, diarrhea, stomach (abdominal) pain, nausea, tiredness, rash and fever.1
Patient Support: GBT Source Solutions™
As part of GBT’s commitment to supporting access to care for patients, we have launched GBT Source Solutions, a resource center for patients who have been prescribed Oxbryta by their healthcare provider. GBT Source Solutions will provide support by:
· Reviewing insurance coverage options and explaining benefits.
· Coordinating shipment of Oxbryta and explaining Specialty Pharmacy benefits.
· Helping to pay for treatment with financial and co-pay assistance for eligible patients.
· Helping to stay on treatment with a nurse support team. The nurse support team is there to support product adherence, and not to replace a patient’s treatment plan. They do not provide medical advice or case management services.
Your role
Your organization plays a critical role in supporting patients, and we are very thankful for everything you do for people living with SCD. Your collaboration is critical to helping inform patients about Oxbryta and GBT’s patient support services for patients prescribed Oxbryta.
If a patient is interested in learning more about Oxbryta, they should consult their healthcare professional or visit www.Oxbryta.com.
We have attached in this email several resources. You may share these with patients, caregivers, and other members of your community.
… Oxbryta fact sheet
… Oxbryta patient education brochure
… GBT Source Solutions brochure
Please feel free to reach out with any questions. We look forward to continuing our collaboration to help improve the lives of SCD patients and provide hope to our community.
Warmest regards,
Jung
INDICATION AND IMPORTANT SAFETY INFORMATION
INDICATION
What is OXBRYTA?
OXBRYTA is a prescription medicine used for the treatment of sickle cell disease in adults and children 12 years of age and older.
It is not known if OXBRYTA is safe and effective in children below 12 years of age.
This indication is approved under accelerated approval based on increase in hemoglobin (Hb). Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trial(s).
IMPORTANT SAFETY INFORMATION
Do not take OXBRYTA if you have had an allergic reaction to voxelotor or any of the ingredients in OXBRYTA. See the end of the patient leaflet for a list of the ingredients in OXBRYTA.
If you are receiving exchange transfusions, talk to your healthcare provider about possible difficulties with the interpretation of certain blood tests when taking OXBRYTA.
Before taking OXBRYTA, tell your healthcare provider about all of your medical conditions, including if you:
· have liver problems
· are pregnant or plan to become pregnant. It is not known if OXBRYTA can harm your unborn baby.
· are breastfeeding or plan to breastfeed. It is not known if OXBRYTA can pass into your breastmilk and if it can harm your baby. Do not breastfeed during treatment with OXBRYTA and for at least 2 weeks after the last dose.
Tell your healthcare provider about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements. Some medicines may affect how OXBRYTA works. OXBRYTA may also affect how other medicines work.
What are the possible side effects of OXBRYTA?
OXBRYTA can cause serious side effects, including:
Serious allergic reactions. Tell your healthcare provider or get emergency medical help right away if you get:
· rash
· hives
· shortness of breath
· swelling of the face
The most common side effects of OXBRYTA include:
· headache
· diarrhea
· stomach (abdominal) pain
· nausea
· tiredness
· rash
· fever
These are not all the possible side effects of OXBRYTA.
Call your doctor for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088.
You may also report side effects to Global Blood Therapeutics at 1-833-428-4968 (1-833-GBT-4YOU).
Keep OXBRYTA and all medicines out of the reach of children.
1 Patient Information Leaflet 11 2019
2 USPI 11 2019  

CRISPR Therapeutics and Vertex Announce Positive Safety and Efficacy Data

Nov 19, 2019

CRISPR Therapeutics and Vertex Announce Positive Safety and Efficacy Data From First Two Patients Treated With Investigational CRISPR/Cas9 Gene-Editing Therapy CTX001® for Severe Hemoglobinopathies

-Two patients treated with CTX001 successfully engrafted and demonstrated an initial safety profile consistent with myeloablative busulfan conditioning and autologous hematopoietic stem cell transplant-
-Beta thalassemia: Patient is transfusion independent with total hemoglobin level of 11.9 g/dL and 10.1 g/dL fetal hemoglobin at nine months after CTX001 infusion-
-Sickle cell disease: Patient is free of vaso-occlusive crises with total hemoglobin level of 11.3 g/dL and 46.6% fetal hemoglobin at four months after CTX001 infusion-
-CRISPR Therapeutics will host a conference call today at 8:00 a.m. ET to review these data-
ZUG, Switzerland and CAMBRIDGE, Mass. and BOSTON, Nov. 19, 2019 (GLOBE NEWSWIRE) — CRISPR Therapeutics (NASDAQ: CRSP) and Vertex Pharmaceuticals Incorporated (NASDAQ: VRTX) today announced positive, interim data from the first two patients with severe hemoglobinopathies treated with the investigational CRISPR/Cas9 gene-editing therapy CTX001 in ongoing Phase 1/2 clinical trials. One patient with transfusion-dependent beta thalassemia (TDT) received CTX001 in the first quarter of 2019 and data for this patient reflect nine months of safety and efficacy follow-up. One patient with severe sickle cell disease (SCD) received CTX001 in mid-2019 and data for this patient reflect four months of safety and efficacy follow-up. These studies are ongoing and patients will be followed for approximately two years following infusion. Several additional patients have been enrolled and have had drug product manufactured across the two studies.
Transfusion-Dependent Beta Thalassemia
The patient with TDT has the β0/IVS-I-110 genotype and required 16.5 transfusions per year (annualized rate during the two years prior to consenting for the study) before enrolling in the clinical study. The patient achieved neutrophil engraftment 33 days after CTX001 infusion and platelet engraftment 37 days after infusion. Two serious adverse events (SAEs) occurred, neither of which the principal investigator (PI) considered related to CTX001: pneumonia in the presence of neutropenia and veno-occlusive liver disease attributed to busulfan conditioning; both subsequently resolved. At nine months after CTX001 infusion, the patient was transfusion independent and had total hemoglobin levels of 11.9 g/dL, 10.1 g/dL fetal hemoglobin, and 99.8% F-cells (erythrocytes expressing fetal hemoglobin).
Sickle Cell Disease
The patient with SCD experienced seven vaso-occlusive crises (VOCs) per year (annualized rate during the two years prior to consenting for the study) before enrolling in the clinical study. The patient achieved neutrophil and platelet engraftment 30 days after CTX001 infusion. Three SAEs occurred, none of which the PI considered related to CTX001: sepsis in the presence of neutropenia, cholelithiasis, and abdominal pain, all of which resolved. At four months after CTX001 infusion, the patient was free of VOCs and had total hemoglobin levels of 11.3 g/dL, 46.6% fetal hemoglobin, and 94.7% F-cells (erythrocytes expressing fetal hemoglobin).
“We are very encouraged by these preliminary data, the first such data to be reported for patients with beta thalassemia and sickle cell disease treated with our CRISPR/Cas9 edited autologous hematopoietic stem cell candidate, CTX001,” said Samarth Kulkarni, Ph.D., Chief Executive Officer of CRISPR Therapeutics. “These data support our belief in the potential of our therapies to have meaningful benefit for patients following a one-time intervention. We continue to enroll these studies as we drive forward to develop CRISPR/Cas9 therapies as a new class of transformative medicines to treat serious diseases.”
“The data we announced today are remarkable and demonstrate that CTX001 has the potential to be a curative CRISPR/Cas9-based gene-editing therapy for people with sickle cell disease and beta thalassemia,” said Jeffrey Leiden, M.D., Ph.D., Chairman, President and Chief Executive Officer of Vertex. “While the data are exciting, we are still in the early phase of this clinical program. We look forward to continuing to work with physicians, patients, caregivers and families over the coming months and years to bring forward the best possible therapy for these two serious diseases and to continue to accelerate our gene-editing programs for other serious diseases such as Duchenne muscular dystrophy and myotonic dystrophy type 1.”
About the Phase 1/2 Study in Transfusion-Dependent Beta Thalassemia
The ongoing Phase 1/2 open-label trial, CLIMB-Thal-111, is designed to assess the safety and efficacy of a single dose of CTX001 in patients ages 18 to 35 with TDT. The study will enroll up to 45 patients and follow patients for approximately two years after infusion. Each patient will be asked to participate in a long-term follow-up study. Enrollment is ongoing at six clinical trial sites in the United States, Canada and Europe.
About the Phase 1/2 Study in Sickle Cell Disease
The ongoing Phase 1/2 open-label trial, CLIMB-SCD-121, is designed to assess the safety and efficacy of a single dose of CTX001 in patients ages 18 to 35 with severe SCD. The study will enroll up to 45 patients and follow patients for approximately two years after infusion. Each patient will be asked to participate in a long-term follow-up study. Enrollment is ongoing at 12 clinical trial sites in the United States, Canada and Europe.
About the Gene–Editing Process in These Trials
Patients who enroll in these studies will have hematopoietic stem and progenitor cells collected from peripheral blood. The patient’s cells will be edited using the CRISPR/Cas9 technology. The edited cells, CTX001, will then be infused back into the patient as part of a stem cell transplant, a process which involves, among other things, a patient being treated with myeloablative busulfan conditioning. Patients undergoing stem cell transplants may also encounter side effects (ranging from mild to severe) that are unrelated to the administration of CTX001. Patients will initially be monitored to determine when the edited cells begin to produce mature blood cells, a process known as engraftment. After engraftment, patients will continue to be monitored to track the impact of CTX001 on multiple measures of disease.
CRISPR Therapeutics Conference Call and Webcast
CRISPR Therapeutics will host a conference call and webcast today at 8:00 a.m. ET. The webcast and presentation will be made available on the CRISPR Therapeutics website at https://crisprtx.gcs-web.com/events in the Investors section under Events and Presentations. Following the live audio webcast, a replay will be available on the Company’s website for approximately 30 days.
Dial-In Information
Live (U.S. / Canada): (800) 895-3361
Live (International): (785) 424-1062
Conference ID: 87198237
About CTX001
CTX001 is an investigational ex vivo CRISPR gene-edited therapy that is being evaluated for patients suffering from TDT or severe SCD in which a patient’s hematopoietic stem cells are engineered to produce high levels of fetal hemoglobin (HbF; hemoglobin F) in red blood cells. HbF is a form of the oxygen-carrying hemoglobin that is naturally present at birth and is then replaced by the adult form of hemoglobin. The elevation of HbF by CTX001 has the potential to alleviate transfusion requirements for TDT patients and painful and debilitating sickle crises for SCD patients.
CTX001 is being developed under a co-development and co-commercialization agreement between CRISPR Therapeutics and Vertex.
About the CRISPR-Vertex Collaboration
CRISPR Therapeutics and Vertex entered into a strategic research collaboration in 2015 focused on the use of CRISPR/Cas9 to discover and develop potential new treatments aimed at the underlying genetic causes of human disease. CTX001 represents the first treatment to emerge from the joint research program. CRISPR Therapeutics and Vertex will jointly develop and commercialize CTX001 and equally share all research and development costs and profits worldwide.
About CRISPR Therapeutics
CRISPR Therapeutics is a leading gene editing company focused on developing transformative gene-based medicines for serious diseases using its proprietary CRISPR/Cas9 platform. CRISPR/Cas9 is a revolutionary gene editing technology that allows for precise, directed changes to genomic DNA. CRISPR Therapeutics has established a portfolio of therapeutic programs across a broad range of disease areas including hemoglobinopathies, oncology, regenerative medicine and rare diseases. To accelerate and expand its efforts, CRISPR Therapeutics has established strategic collaborations with leading companies including Bayer AG, Vertex Pharmaceuticals and ViaCyte, Inc.CRISPR Therapeutics AG is headquartered in Zug, Switzerland, with its wholly-owned U.S. subsidiary, CRISPR Therapeutics, Inc., and R&D operations based in Cambridge, Massachusetts, and business offices in London, United Kingdom. For more information, please visit www.crisprtx.com.
CRISPR Therapeutics Forward-Looking Statement
This press release may contain a number of “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including statements regarding CRISPR Therapeutics’ expectations about any or all of the following: (i) the safety, efficacy and clinical progress of CRISPR Therapeutics’ CTX001 clinical program; (ii) the status and scope of ongoing and potential future clinical trials (including, without limitation, the timing of filing of clinical trial applications and INDs, any approvals thereof and the timing of commencement of clinical trials), development timelines and discussions with regulatory authorities related to product candidates under development by CRISPR Therapeutics and its collaborators; (iii) the number of patients that will be evaluated, the anticipated date by which enrollment will be completed and the data that will be generated by ongoing and planned clinical trials, and the ability to use that data for the design and initiation of further clinical trials; v(iv) the intellectual property coverage and positions of CRISPR Therapeutics, its licensors and third parties; (v) the sufficiency of CRISPR Therapeutics’ cash resources; and (vi) the therapeutic value, development, and commercial potential of CRISPR/Cas9 gene editing technologies and therapies. Without limiting the foregoing, the words “believes,” “anticipates,” “plans,” “expects” and similar expressions are intended to identify forward-looking statements. You are cautioned that forward-looking statements are inherently uncertain. Although CRISPR Therapeutics believes that such statements are based on reasonable assumptions within the bounds of its knowledge of its business and operations, forward-looking statements are neither promises nor guarantees and they are necessarily subject to a high degree of uncertainty and risk. Actual performance and results may differ materially from those projected or suggested in the forward-looking statements due to various risks and uncertainties. These risks and uncertainties include, among others: the potential for initial and preliminary data from any clinical trial (including CTX001) not to be indicative of final trial results; the risk that the initial data from a limited number of patients (as is the case with CTX001 at this time) may not be indicative of results from the full planned study population; the outcomes for each CRISPR Therapeutics’ planned clinical trials and studies may not be favorable; that one or more of CRISPR Therapeutics’ internal or external product candidate programs will not proceed as planned for technical, scientific or commercial reasons; that future competitive or other market factors may adversely affect the commercial potential for CRISPR Therapeutics’ product candidates; uncertainties inherent in the initiation and completion of preclinical studies for CRISPR Therapeutics’ product candidates; availability and timing of results from preclinical studies; whether results from a preclinical trial will be predictive of future results of the future trials; uncertainties about regulatory approvals to conduct trials or to market products; uncertainties regarding the intellectual property protection for CRISPR Therapeutics’ technology and intellectual property belonging to third parties, and the outcome of proceedings (such as an interference, an opposition or a similar proceeding) involving all or any portion of such intellectual property; and those risks and uncertainties described under the heading “Risk Factors” in CRISPR Therapeutics’ most recent annual report on Form 10-K, and in any other subsequent filings made by CRISPR Therapeutics with the U.S. Securities and Exchange Commission, which are available on the SEC’s website at www.sec.gov. Existing and prospective investors are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date they are made. CRISPR Therapeutics disclaims any obligation or undertaking to update or revise any forward-looking statements contained in this press release, other than to the extent required by law.
About Vertex
Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases. The company has four approved medicines that treat the underlying cause of cystic fibrosis (CF) — a rare, life-threatening genetic disease — and has several ongoing clinical and research programs in CF. Beyond CF, Vertex has a robust pipeline of investigational small molecule medicines in other serious diseases where it has deep insight into causal human biology, including pain, alpha-1 antitrypsin deficiency, and APOL1-mediated kidney disease. In addition, Vertex has a rapidly expanding pipeline of genetic and cell therapies for diseases such as sickle cell disease, beta thalassemia, Duchenne muscular dystrophy and type 1 diabetes mellitus.
Founded in 1989 in Cambridge, Mass., Vertex’s global headquarters is now located in Boston’sInnovation District and its international headquarters is in London, UK. Additionally, the company has research and development sites and commercial offices in North America, Europe, Australia and Latin America. Vertex is consistently recognized as one of the industry’s top places to work, including 10 consecutive years on Science magazine’s Top Employers list and top five on the 2019 Best Employers for Diversity list by Forbes. For company updates and to learn more about Vertex’s history of innovation, visit www.vrtx.com or follow us on Facebook, Twitter, LinkedIn, YouTube and Instagram.
(VRTX-GEN)
Vertex Special Note Regarding Forward-Looking Statements
This press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, including, without limitation, the information provided regarding the status of, and expectations with respect to, the CTX001 clinical development program. While Vertex believes the forward-looking statements contained in this press release are accurate, these forward-looking statements represent the company’s beliefs only as of the date of this press release, and there are a number of factors that could cause actual events or results to differ materially from those indicated by such forward-looking statements. Those risks and uncertainties include that the development of CTX001 may not proceed due to safety, efficacy or other reasons, and other risks listed under Risk Factors in Vertex’s annual report and quarterly reports filed with the Securities and Exchange Commission and available through the company’s website at www.vrtx.com. Vertex disclaims any obligation to update the information contained in this press release as new information becomes available.
CRISPR Therapeutics Investor Contact:
Susan Kim, +1 617-307-7503
susan.kim@crisprtx.com
CRISPR Therapeutics Media Contact:
Jennifer Paganelli
WCG on behalf of CRISPR
+1 347-658-8290
jpaganelli@wcgworld.com
Vertex Pharmaceuticals Incorporated
Investors:
Michael Partridge, +1 617-341-6108
or
Zach Barber, +1 617-341-6470
or
Leah Gibson, +1 617-961-1507
Media: mediainfo@vrtx.com
or
North America:
Heather Nichols, +1 617-341-6992
Heather_Nichols@vrtx.com

 

Novartis Adakveo approved by FDA for Sickle Cell Disease


New Novartis medicine Adakveo® (crizanlizumab-tmca) approved by FDA to reduce frequency of pain crises in individuals living with sickle cell disease

  • Sickle cell pain crises are unpredictable, severe events associated with life-threatening complications1
  • Adakveo reduced the annual rate of sickle cell pain crises by 45% compared to placebo (1.63 vs 2.98) and the annual rate of days hospitalized (4 vs 6.87) in a 52-week study2
  • Approximately 100,000 people in the United States, most of whom are of African descent, have sickle cell disease3
  • Approval comes approximately two months ahead of FDA’s priority review action date, allowing Adakveo to be available to patients more quickly

East Hanover, NJ, November 15, 2019 – Novartis announced today that the US Food and Drug Administration (FDA) approved Adakveo® (crizanlizumab-tmca), previously known as SEG101, to reduce the frequency of vaso-occlusive crises (VOCs), or pain crises, in adult and pediatric patients aged 16 years and older with sickle cell disease.4 Adakveo represents the first FDA-approved medicine in sickle cell disease that binds to P-selectin – a cell adhesion protein that plays a central role in the multicellular interactions that can lead to vaso-occlusion.5,6 The medicine is expected to be available to patients in the coming weeks.
The FDA’s decision to approve Adakveo 5 mg/kg is based on results of the 52-week, randomized, placebo-controlled SUSTAIN trial, which showed that Adakveo significantly lowered the median annual rate of VOCs to 1.63 vs 2.98 compared to placebo (P=.010), which is equivalent to a 45% reduction. Reductions in the frequency of VOCs were observed among patients regardless of sickle cell disease genotype and/or hydroxyurea use.2,4
“We know this drug can decrease the frequency of sickle cell pain crises in a significant and clinically meaningful way,” said Kenneth Ataga, MD, Director, Center for Sickle Cell Disease, University of Tennessee Health Science Center at Memphis, and Principal Investigator of the SUSTAIN trial. “The approval of crizanlizumab is an important advancement for people living with this very difficult condition.”
Additional results from the SUSTAIN study include:4

  • A decrease in the median annual rate of days hospitalized to 4 vs 6.87 days when compared with placebo (a 42% reduction)
  • Thirty-six percent of patients treated with Adakveo did not experience a VOC, compared to 17% of placebo-treated patients
  • The median time to first VOC was 4.1 for Adakveo vs 1.4 months for placebo

The most common adverse reactions (incidence > 10%) were nausea (18%), arthralgia (18%), back pain (15%) and pyrexia (11%).4
“The approval of Adakveo marks a new era in the treatment of sickle cell disease, a genetic condition that places an extraordinary burden of unpredictable pain crises on patients and their families,” said Susanne Schaffert, PhD, President, Novartis Oncology. “The stories we have heard from patients about their sickle cell pain crises are devastating. We are pleased to help reimagine medicine together with the sickle cell community and offer new hope for fewer VOCs.”
Considered the clinical hallmark of the disease, sickle cell pain crises are triggered, in part, by multicellular interactions that form clusters of cells, which can block or reduce the blood flow to organs.1,7 Sickle cell pain crises can be frequent and sudden, and are associated with an increased risk of life-threatening complications.1 They also are the main reason why individuals living with sickle cell disease go to the emergency room and are admitted to the hospital.7
“Patients with sickle cell disease often face unique challenges, and have long suffered silently through unimaginable pain crises,” said Beverley Francis-Gibson, President and CEO of the Sickle Cell Disease Association of America. “We are excited to have a new medicine that may help many of the thousands of people living with sickle cell disease by reducing the frequency of these potentially dangerous and painful episodes.”
About Sickle Cell Disease
Sickle cell disease is a complex and debilitating genetic blood disorder that goes beyond sickle-shaped red blood cells. The disease is associated with chronic inflammation, causing higher levels of cell adhesion proteins, including P-selectin, which make both the blood vessels and certain blood cells stickier and prone to multicellular interactions, or clusters, in the bloodstream. This environment can lead to the acute episodes of pain known as sickle cell pain crises, or VOCs, as well as life-threatening complications.1,7,8 VOCs are the main reason why individuals living with sickle cell disease seek medical care in hospitals,7 leading to approximately 200,000 ER visits in the US every year.9,10
Approximately 100,000 people in the US have sickle cell disease.3 People of African ancestry make up 90% of the population with sickle cell disease in the US. However, sickle cell disease is also prevalent among people of Hispanic, South Asian, Southern European, and Middle Eastern ancestry. Sickle cell disease occurs in about 1 in 365 and 1 in 16,300 African-American and Hispanic-American births, respectively.3
About Adakveo
Adakveo® (crizanlizumab-tmca) – previously known as SEG101 – is indicated to reduce the frequency of VOCs, or pain crises, in adults and pediatric patients aged 16 years and older with sickle cell disease. It is the first and only targeted biologic that works by binding to P-selectin, a cell adhesion protein that plays a central role in the multicellular interactions that can lead to vaso-occlusion in sickle cell disease.
By binding to P-selectin on the surface of the activated endothelium and platelets, Adakveo blocks interactions between endothelial cells, platelets, red blood cells, and leukocytes.4
About SUSTAIN
SUSTAIN is a randomized, multicenter, placebo-controlled, double-blind study. A total of 198 patients with any genotype of sickle cell disease (HbSS, HbSC, HbS/beta0-thalassemia, HbS/beta+-thalassemia, and others) and a history of 2-10 VOCs in the previous 12 months were eligible for inclusion. Patients were randomized 1:1:1 to Adakveo 5 mg/kg (N = 67), Adakveo 2.5 mg/kg (N = 66), or placebo (N = 65) administered over a period of 30 minutes by intravenous infusion on Week 0, Week 2, and every 4 weeks thereafter, for a treatment duration of 52 weeks.
The primary efficacy outcome was the annual rate of VOCs leading to a healthcare visit. A VOC leading to a healthcare visit was defined as an acute episode of pain with no cause other than a vaso-occlusive event that required a medical facility visit and treatment with oral or parenteral opioids, or parenteral NSAIDs. Acute chest syndrome, hepatic sequestration, splenic sequestration, and priapism (requiring a visit to a medical facility) were also considered VOCs. Key secondary and other efficacy endpoints include annual rate of days hospitalized, time to first VOC leading to healthcare visit, and number of patients that did not experience a VOC.
Patient Access and Support
Novartis is committed to helping ensure that our medicines are accessible to as many patients as possible. With the approval of Adakveo in the United States, we now offer resources and support to address a range of needs. Adakveo Support at PANO (Patient Assistance Now Oncology) is a support center staffed by insurance specialists and case managers who can help eligible patients start and stay on treatment. Dedicated support specialists are available to help direct callers to services that best fit their needs. Patients or providers can call 800-282-7630 or visit Patient.NovartisOncology.com or HCP.Novartis.com/Access to learn more about eligibility and to enroll.
Novartis Commitment to Sickle Cell Disease in Africa
Sickle cell is a global disease and is most widespread in sub-Saharan Africa. Unfortunately, we can see a clear disparity when comparing Africa with other parts of the world, where sickle cell is often managed as a chronic disease. Building on years of engagement in Africa, working to reduce the impact of malaria and other conditions, Novartis is taking steps to help address the needs of sickle cell patients as well, beginning in Ghana. Our partnership with the Ghana Ministry of Health, the Ghana Health Service, and the Sickle Cell Foundation of Ghana aims to improve the diagnosis and treatment of people with sickle cell disease through a comprehensive approach to screening and diagnosis, treatment and disease management, training and education, and elevating basic and clinical research and scientific capabilities. These activities include facilitating access to high-quality hydroxyurea and other basic medicines to enhance the standard of care.
To date, Novartis has delivered more than 20,000 hydroxyurea treatments to Ghana, with plans to deliver a total of 60,000 treatments by the end of the year. In addition, Novartis is developing a child-friendly formulation of hydroxyurea and is committed to implementing two clinical trials with crizanlizumab in Ghana and Kenya – an important step to bringing this innovative medicine to patients. Crizanlizumab trials in Africa are expected to start in 2020.
Indication
Adakveo® (crizanlizumab-tmca) is used in people 16 years of age and older, who have sickle cell disease, to help reduce how often certain episodes of pain (crises) happen. It is not known if Adakveo is safe and effective in children under 16 years of age.
Important Safety Information
Adakveo may cause serious side effects, including infusion reactions. Infusion reactions may happen within 24 hours of receiving an infusion of Adakveo. Patients should tell their health care provider right away if they get any of the following signs and symptoms of an infusion reaction such as fever, chills or shivering, nausea, vomiting, tiredness, dizziness, sweating, hives, itching, or shortness of breath or wheezing. Health care providers may monitor their patients for signs and symptoms of infusion reactions.
Adakveo may interfere with automated platelet counts (platelet clumping). Patients should tell their health care provider that they are receiving Adakveo before having any blood tests. Health care providers should run blood samples as soon as possible or use tubes containing citrate.
Before receiving Adakveo, patients should tell their health care provider if they are pregnant or plan to become pregnant. It is not known if Adakveo may harm an unborn baby.
The most common side effects (incidence ≥10%) include nausea, back pain, joint pain, and fever.
Please see full Prescribing Information for Adakveo at https://wpdemo12.com/wp-content/uploads/2024/01/adakveo.pdf.
Disclaimer
This press release contains forward-looking statements within the meaning of the United States Private Securities Litigation Reform Act of 1995. Forward-looking statements can generally be identified by words such as “potential,” “can,” “will,” “plan,” “expect,” “anticipate,” “look forward,” “believe,” “committed,” “investigational,” “pipeline,” “launch,” or similar terms, or by express or implied discussions regarding potential marketing approvals, new indications or labeling for the investigational or approved products described in this press release, or regarding potential future revenues from such products. You should not place undue reliance on these statements. Such forward-looking statements are based on our current beliefs and expectations regarding future events, and are subject to significant known and unknown risks and uncertainties. Should one or more of these risks or uncertainties materialize, or should underlying assumptions prove incorrect, actual results may vary materially from those set forth in the forward-looking statements. There can be no guarantee that the investigational or approved products described in this press release will be submitted or approved for sale or for any additional indications or labeling in any market, or at any particular time. Nor can there be any guarantee that such products will be commercially successful in the future. In particular, our expectations regarding such products could be affected by, among other things, the uncertainties inherent in research and development, including clinical trial results and additional analysis of existing clinical data; regulatory actions or delays or government regulation generally; global trends toward health care cost containment, including government, payor and general public pricing and reimbursement pressures and requirements for increased pricing transparency; our ability to obtain or maintain proprietary intellectual property protection; the particular prescribing preferences of physicians and patients; general political and economic conditions; safety, quality or manufacturing issues; potential or actual data security and data privacy breaches, or disruptions of our information technology systems, and other risks and factors referred to in Novartis AG’s current Form 20-F on file with the US Securities and Exchange Commission. Novartis is providing the information in this press release as of this date and does not undertake any obligation to update any forward-looking statements contained in this press release as a result of new information, future events or otherwise.
About Novartis
Novartis is reimagining medicine to improve and extend people’s lives. As a leading global medicines company, we use innovative science and digital technologies to create transformative treatments in areas of great medical need. In our quest to find new medicines, we consistently rank among the world’s top companies investing in research and development. Novartis products reach more than 750 million people globally and we are finding innovative ways to expand access to our latest treatments. About 109,000 people of more than 140 nationalities work at Novartis around the world. Novartis Pharmaceuticals Corporation, a US affiliate of Novartis, is located in East Hanover, NJ. Find out more at www.novartis.com.
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References

  1. Steinberg M. Management of sickle cell disease. N Engl J Med. 1999;340(13):1021-1030.
  2. Ataga KI, Kutlar A, Kanter J et al. Crizanlizumab for the prevention of pain crises in sickle cell disease. N Engl J Med. 2017;376(5):429-439.
  3. American Society of Hematology. State of sickle cell disease 2016 report. http://www.scdcoalition.org/pdfs/ASH%20State%20of%20Sickle%20Cell%20Disease%202016%20Report.pdf. Accessed October 24, 2019.
  4. Adakveo (crizanlizumab) prescribing information. East Hanover, New Jersey, USA. Novartis Pharmaceuticals Corporation; November 2019.
  5. Rees DC, Williams TN, Gladwin MT. Sickle-cell disease. Lancet. 2010;376(9757):2018-2031.
  6. Lawrence MB, Springer TA. Leukocytes roll on a selectin at physiologic flow rates: distinction from and prerequisite for adhesion through integrins. Cell. 1991;65(5):859-873.
  7. Gutsaeva D, Parkerson J, Yerigenahally S, et al. Inhibition of cell adhesion by anti–P-selectin aptamer: a new potential therapeutic agent for sickle cell disease. Blood. 2011;117(2):727-735.
  8. Sparkenbaugh E, Pawlinski R. Interplay between coagulation and vascular inflammation in sickle cell disease. Br J Haematol. 2013;162(1):1-22.
  9. Ballas SK, Gupta K, Adams-Graves P. Sickle cell pain: a critical reappraisal. Blood. 2012;120(18):3647-3656.
  10. Yusuf HR, Atrash HK, Grosse SD, Parker CS, Grant AM. Emergency department visits made by patients with sickle cell disease: a descriptive study, 1999-2007.Am J Prev Med. 2010;38(Suppl):S536-S541.

Novartis Global External Communications
E-mail: media.relations@novartis.com  

Donate Blood to Support Sickle Cell Patients

September is Sickle Cell Awareness Month; Red Cross blood and platelet donations needed for patients
This school year students battling sickle cell disease and childhood cancers prepare to face challenges in the classroom unknown to their healthy peers. Many will fall behind in coursework after missing weeks of school, require tutoring and special education services. But there is a way people can help ease the struggle of these serious diseases.
September is Sickle Cell Awareness Month, making it a great time to donate blood. The Red Cross, Sickle Cell Disease Association of America, Inc., and the Sickle Cell Foundation of Georgia, Inc. formed a partnership last year to increase blood donations to support the SCD community. Over the five-year partnership, the goal is to collect 15,000 blood donations. Donations like these are essential to students living with SCD who rely heavily on lifesaving blood and platelet donations from generous and diverse donors, to help keep them in the classroom. Schedule an appointment to give blood with the American Red Cross by visiting RedCrossBlood.org, using the Red Cross Blood Donor App, calling 1-800-RED-CROSS or activating the Blood Scheduling Skill for Amazon Alexa.
“Sickle cell disease and childhood cancer profoundly impact the lives of young people— ensuring there is a diverse and sufficient blood supply is critical to their survival and ability to get back to school,” said Dr. Yvette Marie Miller, executive medical director at the Red Cross. “The Red Cross encourages diverse blood donors to roll up a sleeve this September and throughout the year to help the many diverse patients in need of blood each day.”
Blood type, like eye color, is an inherited trait passed genetically from parents. The vast majority of blood types fall into one of the major ABO groups, but for some patients with rare blood types, blood must be matched closely, beyond the primary A, B, O and AB blood types, to reduce the risk of developing complications from transfusion therapy. A patient in need is more likely to find a compatible blood match from a donor of the same race or ethnicity.
Students with sickle cell disease admit it impacts performance
A recent study conducted by researchers from Cincinnati Children’s Hospital Medical Center and University of Cincinnati College of Medicine indicates that 60% of participants reported that sickle cell disease interfered with their school performance and over 75% of participants diagnosed with the most severe sickle cell form reported receiving special education services due to absences.
Regular blood transfusions are one of the most common treatments for sickle cell disease, which have been proven to help patients by unblocking blood vessels, alleviating pain from a sickle cell crisis. Many patients require transfusions multiple times per year throughout their lifetime.
Braden Green suffered from Acute Chest Syndrome, the leading cause of death in sickle cell patients and needed blood transfusions to survive. During his recovery he missed six weeks of school and depended on instructors to help him get caught up.
His mom, Brenda Green, recalls watching the donated blood enter her son’s body. “I realized at that moment how important a blood donation is.” She vowed to become an advocate for blood donations and has since hosted several blood drives for patients in need.
“If it hadn’t been for that blood transfusion, I am not sure where we would be today.”
Blood donation information
All blood types are needed to ensure a reliable supply for patients. A blood donor card or driver’s license or two other forms of identification are required at check-in. Individuals who are 17 years of age in most states (16 with parental consent where allowed by state law), weigh at least 110 pounds and are in generally good health may be eligible to donate blood. High school students and other donors 18 years of age and younger also have to meet certain height and weight requirements.
Blood and platelet donors can save time at their next donation by using RapidPass® to complete their pre-donation reading and health history questionnaire online, on the day of their donation, before arriving at the blood drive. To get started, follow the instructions at RedCrossBlood.org/RapidPass or use the Red Cross Blood Donor App.
About the American Red Cross:
The American Red Cross shelters, feeds and provides emotional support to victims of disasters; supplies about 40 percent of the nation’s blood; teaches skills that save lives; provides international humanitarian aid; and supports military members and their families. The Red Cross is a not-for-profit organization that depends on volunteers and the generosity of the American public to perform its mission. For more information, please visit redcross.org or cruzrojaamericana.org, or visit us on Twitter at @RedCross.  

GBT and SCDAA to Host 8th Annual Sickle Cell Disease (SCD) Therapeutics Conference

GlobalBlood Therapeutics, Inc. (GBT) (NASDAQ: GBT) and the Sickle Cell Disease Association of America, Inc. (SCDAA) announced that they will host the 8th Annual Sickle Cell Disease (SCD) Therapeutics Conference on Tuesday, September 10, 2019, at The Westin Washington, D.C. U.S. Representative Robin Kelly, chair of the Congressional Black Caucus Health Braintrust, will deliver the keynote address.
Taking place during National Sickle Cell Awareness Month, the SCD Therapeutics Conference will highlight the latest medical advances and future trends in the treatment of patients with SCD. In addition to Rep. Kelly’s keynote address, the program will feature panel discussions and presentations from leading physicians, patient advocates and healthcare policymakers on a range of issues and topics, including the impact of SCD on the brain and cognitive function and hot topics in SCD from the perspective of patients and caregivers. The Conference will also feature updates from the American Society of Hematology Research Collaborative’s SCD Clinical Trials Network and the Sickle Cell Community Consortium, as well as presentations from biopharmaceutical industry leaders.
“We are honored to host, in partnership with the Sickle Cell Disease Association of America, the annual SCD Therapeutics Conference for the third year in a row, as it will bring together patient advocates, opinion leaders and clinical experts,” said Ted W. Love, M.D., president and chief executive officer of GBT. “Support and collaboration across the broader community is essential as we work together to try to fundamentally transform how SCD is treated. We are optimistic that this year’s Conference will spur important discussions as we continue to pursue our shared mission of making a meaningful difference in the future of SCD care.”
“As co-hosts of the annual SCD Therapeutics Conference, we are thrilled to help advance meaningful discussions aimed at understanding and addressing the needs of the SCD community,” said Beverley Francis-Gibson, B.A., M.A., president and chief executive officer of SCDAA. “It is necessary that we continue to learn and grow based on the latest advancements that will be discussed at the Conference, in addition to understanding future issues and trends for treating patients with SCD.”
The 8th Annual SCD Therapeutics Conference will feature the following speakers and panelists:

  • Biree Andemariam, M.D., Associate Professor, Department of Medicine, University of Connecticut
  • Lakiea Bailey, Ph.D., Founder, Sickle Cell Community Consortium
  • Velvet Brown-Watts, Founder-Chairperson, Supporters of Families with Sickle Cell Disease
  • Chuck Chesson, Ph.D., Director, Sickle Cell Disease Clinical Trials Network
  • Jew-EL Darbone, Sickle Cell Patient Advocate
  • Jeremie Estepp, M.D., Medical Director, Clinical Translation Program in Hematology, St. Jude Children’s Research Hospital
  • Beverley Francis-Gibson, B.A., M.A., President and Chief Executive Officer, SCDAA
  • André Harris, CHW, Sickle Cell Patient Advocate
  • The Honorable Robin Kelly, Chair, Congressional Black Caucus Health Braintrust, United States House of Representatives
  • Allison King, M.D., M.P.H., Ph.D., Associate Professor of Occupational Therapy, Pediatrics, Medicine, Surgery (Prevention and Control) and Education,
  • Washington University School of Medicine
  • Hertz Nazaire, Sickle Cell Patient Advocate
    Additionally, multiple companies that are developing treatments for patients with SCD will present at the Conference. They include Addmedica, CRISPR Therapeutics/Vertex, Cyclerion, Emmaus Life Sciences, GBT, Imara and Sanofi. To ensure that those who are unable to attend the Conference can benefit from the presentations and information sharing, GBT is collaborating with Sickle Cell Warriors, an SCD community organization, to livestream from the Conference on the organization’s Facebook page at www.facebook.com/SickleCellWarriors. Following the event, GBT will post videos on its corporate YouTube channel. For more information about the Conference or to register to attend, visit http://www.scdconference.com.

 

SCDAA Forms International Media Partnership to Spread SCD Awareness


New International Media Partnership Will Enhance Awareness Efforts and Increase Awareness about Sickle Cell Disease and Sickle Cell Trait
Sickle Cell Disease Association of America, Inc. (SCDAA) and the African Sickle Cell News & World Report are proud to announce a new international media partnership that will enhance global awareness efforts about sickle cell disease (SCD). The partnership will use digital platforms and print materials to share educational information with individuals living with SCD and their families and the general public. The partnership will work to dispel myths about the disease, share patient stories and treatment options and engage new audiences.
“Some research estimates that about 240,000 babies are born with sickle cell disease in sub-Saharan Africa every year, and that at least half of such children die before age five,” said SCDAA President and CEO Beverley Francis-Gibson. “Education and early diagnosis is paramount to extending life expectancies and saving lives, and that is why this partnership is so important. African Sickle Cell News & World Report focuses on sharing information about this disease from around the world. Together, we can enhance our efforts to educate and advocate on behalf of those affected by this disease.”
SCD is a global health problem affecting millions of people around the world. It is estimated that approximately 100,000 Americans have the disease, and more than 1,000,000 worldwide have sickle cell trait. Each year, approximately 1,000 babies in the United States are born with SCD, and there is no universal cure for this life-threatening disease.
“We are excited to partner with SCDAA, the leading national voice for the sickle cell community,” said Ayoola Olajide, Editor, African Sickle Cell News & World Report. “Through education, awareness and advocacy, we seek to collaboratively bring worldwide attention to sickle cell disease and to extend SCDAA’s reach internationally. We are looking forward to this partnership and to continuing to support the international sickle cell community in this collaborative initiative.”  

SCDAA Partners with The Pain Community to Support the Sickle Cell Community by Providing Comprehensive Integrative Pain Management Educational Information

Sickle Cell Disease Association of America, Inc. (SCDAA) and The Pain Community are proud to announce a new partnership that will increase education and awareness information about comprehensive integrative pain management and wellness to support individuals living with sickle cell disease (SCD) and their family members. The partnership will use digital platforms and other collateral to share important resources and to engage those affected by SCD in achieving better health and wellness in partnership with their healthcare providers.
“Pain is the most common complication of SCD, and one of main reasons that people with SCD go to the emergency room or hospital,” said SCDAA President and CEO Beverley Francis-Gibson. “The Pain Community shares with SCDAA the goal of improving the quality of life for individuals living with pain, and SCDAA looks forward to working with them to help empower those living with sickle cell disease by providing education and advocacy information for managing pain.”
SCD is a global health problem affecting millions of people around the world. It is estimated that approximately 100,000 Americans have the disease, and more than 1,000,000 worldwide have sickle cell trait. Each year, approximately 1,000 babies in the United States are born with SCD, and there is no universal cure for this life-threatening disease.
“The Pain Community is thrilled to partner with the Sickle Cell Disease Association of America, Inc.,” said TPC Board Chair Karen Keifer, MSN, APN, NP-C, RN-BC. “This affiliation is a great opportunity to amplify SCDAA’s work and share the educational, coping and advocacy resources available that are free at PainCommunity.org We look forward to ensuring that the sickle cell community has resources to help manage their pain and improve their quality of life.”  

GBT and Advocates Launch Disease Awareness Campaign Focused on Breaking Down Stigmas Associated with Sickle Cell Disease

Company Also Launches Disease Awareness Campaign to Raise Awareness Among Physicians About the Silent Damage Caused by Sickle Cell Disease
Global Blood Therapeutics, Inc. (GBT) (NASDAQ: GBT), in partnership with sickle cell community-based organizations (CBOs), today announced the launch of Sickle Cell Speaks, a national campaign focused on breaking down stigmas associated with sickle cell disease (SCD).
Sickle Cell Speaks is designed to bring together a community of people, from patients to caregivers to friends and relatives, whose lives are affected by SCD. The campaign shares the individual experiences of those living with SCD through authentic stories from a diverse group of patients and caregivers. Sickle Cell Speaks aims to inspire hope by showcasing stories of strength and to dispel the misconceptions about SCD.
“The sickle cell patient experience is highly varied, and highlighting the personal stories of these individuals, as well as their family and friends, will help grow understanding and awareness of this condition,” said Beverly Francis-Gibson, president and chief executive officer of the Sickle Cell Disease Association of America. “We are proud to partner with GBT to help break down stigmas and make our voices heard.”
Additional partners of Sickle Cell Speaks include Sickle Cell Community Consortium, Sickle Cell 101, Bold Lips for Sickle Cell and Sickle Cell Warriors.
“We are partnering with several CBOs representing the sickle cell community to educate people about SCD and to help break down the barriers, particularly the misconceptions and social stigmas, that affect how those with SCD are viewed by society and which ultimately impact patients’ ability to access quality care,” said Ted W. Love, M.D., president and chief executive officer of GBT. “In addition to this patient-focused initiative, we recently launched a separate campaign aimed at engaging health care professionals and facilitating discussion around the root cause and molecular basis of the disease. Together, our patient and physician campaigns underscore GBT’s commitment to bringing together a broad range of stakeholders, with the goal of improving the lives of people living with sickle cell.”
GBT’s health care professional-focused disease awareness campaign, SCD Silent Damage, seeks to increase attention among physicians regarding hemoglobin polymerization, the molecular
basis of SCD that initiates the sickling of red blood cells and the ensuing cascade of clinical complications that drive high levels of morbidity and mortality in patients.
SCD is a rare, inherited disease that affects approximately 100,000 people in the United States. People of African descent make up 90 percent of this population, although it also affects people of Hispanic, South Asian, Southern European and Middle Eastern ancestry.1 Red blood cells are responsible for carrying oxygen throughout the body. In people with SCD, the normally round and flexible red blood cells become shaped like crescents because the hemoglobin within them polymerizes to form rods, which deform the cells. These rigid, crescent-shaped cells are not able to pass through small blood vessels and tend to break apart easily, leading to anemia, fatigue, episodes of pain and organ damage.  

BLACKDOCTOR.ORG is a New Media Partner of the Sickle Cell Disease Association of America, Inc.

New National Media Partnership Will Enhance Awareness Efforts and Increase Awareness about Sickle Cell Disease and Sickle Cell Trait
 Sickle Cell Disease Association of America, Inc. (SCDAA) and BLACKDOCTOR.ORG are proud to announce a new media partnership that will enhance national awareness efforts about sickle cell disease (SCD). The partnership will use digital platforms, events and speaking opportunities to share educational information with individuals living with SCD and their families and the general public. The partnership will work to dispel myths about the disease and to share patient stories, treatment options, and initiatives happening within the sickle cell community. The partnership also will engage new audiences, create new media and public relations opportunities and help to break the silence surrounding the disease.
“SCDAA is very excited to announce this media partnership and the new opportunities we have together to help remove stigma and to share stories from those living with sickle cell disease,” said SCDAA President and CEO Beverley Francis-Gibson. “BLACKDOCTOR.ORG provides access to innovative new approaches to health information that helps individuals empower themselves by breaking through the health disparities and gaining better control of their health and well-being. SCDAA looks forward to working with BLACKDOCTOR.ORG to increase awareness about sickle cell disease and to encourage individuals to get involved in our efforts to advocate on behalf of those affected by this disease.”
“SCDAA and its chapters across the country have a long history of serving the sickle cell community through education, awareness and advocacy, and BLACKDOCTOR.ORG is proud to partner with them in our collective goals to empower individuals with their health by raising awareness about sickle cell disease,” said Reginald Ware, BLACKDOCTOR.ORG CEO. “Our media partnership seeks to help SCDAA broaden its reach into the African American community and to get people involved in an important cause. We are looking forward to supporting SCDAA and the sickle cell community in this collaborative initiative.”
About BLACKDOCTOR.ORG
BLACKDOCTOR.ORG is the all-important primary destination that redefines Black health. It aims to be a trusted, daily resource for healthier, happier living, and daily medicine. A Powerful Resource for Black Health BlackDoctor.org is the world’s largest and most comprehensive online health resource specifically targeted to African Americans. BLACKDOCTOR.ORG understands that the uniqueness of Black culture plays a role in our health. BLACKDOCTOR.ORG gives access to innovative new approaches to the health. Guided by the leading physicians, BLACKDOCTOR.ORG provides essential, but difficult to find, health resources, including free referrals to the top Black physicians and health articles that focus on both culturally accurate content and general health topics.  

Novartis Unveils “The Untold Stories of Sickle Cell Disease,” a worldwide patient story project.

On June 19, 2019, for World Sickle Cell Awareness Day, Novartis unveiled “The Untold Stories of Sickle Cell Disease,” a worldwide patient story project.
This unique program offers a powerful glimpse into the lives of people touched by sickle cell disease – how it affects their lives and how they work to overcome it. You can see and view the stories at www.UntoldSickleCellStories.com, along with disease education information.
You also can check out a 3-minute worldwide promo video at https://app.box.com/s/idrvk9nrr1fh97x5gr5ymw7iltpd2npa