Press Release
GBT Announces Plans to Seek Regulatory Approval for Oxbryta® (voxelotor) to Treat Sickle Cell Patients in Europe
Intends to seek full marketing authorization for Oxbryta to treat hemolytic anemia in sickle cell disease
Marketing Authorization Application submission planned by mid-2021
An oral, once daily therapy, Oxbryta directly inhibits hemoglobin polymerization, the root cause of the sickling and destruction of red blood cells in SCD. The sickling process causes hemolytic anemia (low hemoglobin due to red blood cell destruction), which impairs adequate oxygen delivery to the tissues and organs in the body.
“Anemia and hemolysis are well established to cause morbidity and mortality in sickle cell disease. At GBT, our focus is to address sickle cell disease at its core and modify the course of the disease, with the goal of mitigating the serious and life-threatening complications that often lead to long-term damage and early death,” said
Based on recent discussions with the EMA, GBT intends to seek full marketing authorization of Oxbryta. The planned MAA will include data from the Phase 3 HOPE (Hemoglobin Oxygen Affinity Modulation to Inhibit HbS PolymErization) Study and the Phase 2 HOPE-KIDS 1 Study, both of which enrolled patients at clinical sites in
In
Oxbryta received
About Sickle Cell Disease
Sickle cell disease (SCD) affects an estimated 100,000 people in
About Oxbryta® (voxelotor) Tablets
Oxbryta (voxelotor) is an oral, once-daily therapy for patients with sickle cell disease (SCD). Oxbryta works by increasing hemoglobin’s affinity for oxygen. Since oxygenated sickle hemoglobin does not polymerize, GBT believes Oxbryta blocks polymerization and the resultant sickling and destruction of red blood cells, which are primary pathologies faced by every single person living with SCD. With the potential to improve hemolytic anemia and oxygen delivery, GBT believes that Oxbryta has the potential to modify the course of SCD. On November 25, 2019, Oxbryta received U.S. Food and Drug Administration (FDA) accelerated approval for the treatment of SCD in adults and children 12 years of age and older.2 As a condition of accelerated approval, GBT will continue to study Oxbryta in the HOPE-KIDS 2 Study, a post-approval confirmatory study using transcranial Doppler (TCD) flow velocity to assess the ability of the therapy to decrease stroke risk in children 2 to 15 years of age.
In recognition of the critical need for new SCD treatments, the FDA granted Oxbryta Breakthrough Therapy, Fast Track, Orphan Drug, and Rare Pediatric Disease designations for the treatment of patients with SCD. Additionally, Oxbryta has been granted Priority Medicines (PRIME) designation from the
Important Safety Information
Oxbryta should not be taken if the patient has 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. Oxbryta can cause serious side effects, including serious allergic reactions. Patients should tell their healthcare provider or get emergency medical help right away if they get rash, hives, shortness of breath, or swelling of the face.
Patients receiving exchange transfusions should talk to their healthcare provider about possible difficulties with the interpretation of certain blood tests when taking Oxbryta.
The most common side effects of Oxbryta include headache, diarrhea, stomach (abdominal) pain, nausea, tiredness, rash, and fever. These are not all the possible side effects of Oxbryta. Before taking Oxbryta, patients should tell their healthcare provider about all medical conditions, including if they have liver problems; if they are pregnant or plan to become pregnant as it is not known if Oxbryta can harm an unborn baby; or if they are breastfeeding or plan to breastfeed as it is not known if Oxbryta can pass into breastmilk or if it can harm a baby. Patients should not breastfeed during treatment with Oxbryta and for at least 2 weeks after the last dose.
Patients should tell their healthcare provider about all the medicines they 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.
Patients are advised to call their doctor for medical advice about side effects. Side effects can be reported to FDA at 1-800-FDA-1088. Side effects can also be reported to Global Blood Therapeutics at 1-833-428-4968 (1-833-GBT-4YOU).
Full Prescribing Information for Oxbryta is available at Oxbryta.com.
About Global Blood Therapeutics
Global Blood Therapeutics (GBT) is a biopharmaceutical company dedicated to the discovery, development, and delivery of life-changing treatments that provide hope to underserved patient communities. Founded in 2011, GBT is delivering on its goal to transform the treatment and care of sickle cell disease (SCD), a lifelong, devastating inherited blood disorder. The company has introduced Oxbryta® (voxelotor), the first FDA-approved treatment that directly inhibits sickle hemoglobin polymerization, the root cause of red blood cell sickling in SCD. GBT is also advancing its pipeline program in SCD with inclacumab, a p-selectin inhibitor in development to address pain crises associated with the disease. In addition, GBT’s drug discovery teams are working on new targets to develop the next generation of treatments for SCD. To learn more, please visit www.gbt.com and follow the company on Twitter @GBT_news.
Forward-Looking Statements
Certain statements in this press release are forward-looking within the meaning of the Private Securities Litigation Reform Act of 1995, including statements containing the words “will,” “anticipates,” “plans,” “believes,” “forecast,” “estimates,” “expects,” and “intends,” or similar expressions. These forward-looking statements are based on GBT’s current expectations and actual results could differ materially. Statements in this press release may include statements that are not historical facts and are considered forward-looking within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. GBT intends these forward-looking statements, including statements regarding GBT’s priorities, dedication, focus, goals, and vision; the safety, efficacy, and mechanism of action of Oxbryta and other product characteristics; the commercialization, delivery, availability, use, and commercial and medical potential of Oxbryta; ongoing and planned studies of Oxbryta and related protocols, activities, and expectations; the potential regulatory approval for Oxbryta to treat patients in
References
European Medicines Agency . https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu3182125. AccessedJune 12, 2020 .- Oxbryta (voxelotor) tablets prescribing information. South San Francisco, Calif.
Global Blood Therapeutics, Inc. ; November 2019. - Centers for Disease Control and Prevention website. Sickle Cell Disease (SCD). https://www.cdc.gov/ncbddd/sicklecell/data.html. Accessed June 3, 2019.
- National Heart, Lung, and Blood Institute website. Sickle Cell Disease. https://www.nhlbi.nih.gov/health-topics/sickle-cell-disease. Accessed August 5, 2019.
- Rees DC, et al.
Lancet . 2010;376(9757):2018-2031. - Kato GJ, et al. Nat Rev Dis Primers. 2018;4:18010.
- Kato GJ, et al. J Clin Invest. 2017;127(3):750-760.
- Caboot JB, et al. Paediatr Respir Rev. 2014;15(1):17-23.
Contact Information:
650-410-3258
simmergut@gbt.com
650-741-7730
syao@gbt.com
Source: Global Blood Therapeutics, Inc.