“There are up to 100,000 people estimated to have sickle cell disease in
A first-in-class oral, once-daily therapy, voxelotor directly inhibits hemoglobin polymerization, the root cause of the sickling and destruction of red blood cells in SCD. The sickling process causes anemia and hemolysis (low hemoglobin due to red blood cell destruction), which impairs adequate oxygen delivery to tissues and organs in the body. Voxelotor is not currently approved for marketing by ANVISA in Brazil.
An expanded access program is a mechanism to make medicines available pre-approval upon request by a physician for eligible patients with no alternative treatment option. ANVISA’s authorization of an EAP is based on the following criteria:
- The product is intended for patients with a serious, debilitating, and/or life-threatening disease.
- There is no satisfactory therapeutic alternative with products registered in
Brazil . - The authorization to use the product is delivered based on the request, and only under the responsibility of the prescribing physician, as it is expected to deliver a significant benefit to the patient.
Under the voxelotor EAP, voxelotor will be supplied free of charge for as long as the patient requires treatment. This program is being implemented in partnership with and administered by Inceptua Group’s Medicines Access division, which has expertise in the strategy, design, and operational implementation of pre-approval access programs that make pharmaceutical products available to patients as appropriate in multiple countries around the world.
About Sickle Cell Disease
It is estimated that more than 100,000 people in the
About Voxelotor
Voxelotor is an oral, once-daily therapy for patients with sickle cell disease (SCD). Voxelotor works by increasing hemoglobin’s affinity for oxygen. Since oxygenated sickle hemoglobin does not polymerize, voxelotor inhibits sickle hemoglobin polymerization and the resultant sickling and destruction of red blood cells leading to hemolysis and anemia, which are primary pathologies faced by every single person living with SCD. Through addressing hemolysis and anemia and improving oxygen delivery throughout the body, GBT believes that voxelotor has the potential to modify the course of SCD.
In November 2019, the U.S. Food and Drug Administration (FDA) granted accelerated approval for voxelotor tablets, under the brand name Oxbryta®, for the treatment of SCD in adults and children 12 years of age and older, and in December 2021, the FDA expanded the approved use of Oxbryta for the treatment of SCD in patients 4 years of age and older in the United States.10 As a condition of accelerated approval for patients ages 4 and older in the United States, GBT is studying 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 14 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 received the prestigious 2021 Prix Galien USA award for “Best Biotechnology Product” from The Galien Foundation.
Please click here for Important Safety Information and full Prescribing Information, including Patient Information for Oxbryta in the U.S.
About
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, starting with sickle cell disease (SCD). Founded in 2011, GBT is delivering on its goal to transform the treatment and care of SCD, a lifelong, devastating inherited blood disorder. The company has introduced Oxbryta (voxelotor), the first FDA-approved medicine that directly inhibits sickle hemoglobin (HbS) 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 Phase 3 development to address pain crises associated with the disease, and GBT021601 (GBT601), the company’s next generation HbS polymerization inhibitor. In addition, GBT’s drug discovery teams are working on new targets to develop the next wave of potential 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, commitment, focus, goals, mission, vision and positioning; safety, efficacy and mechanism of action of Oxbryta (or voxelotor) and other product characteristics; commercialization, delivery, availability, use and commercial and medical potential of Oxbryta; the expanded access program for Oxbryta in
References
Centers for Disease Control and Prevention . Sickle Cell Disease Data and Statistics (SCD). https://www.cdc.gov/ncbddd/sicklecell/data.html. AccessedJune 7, 2022 .European Medicines Agency . https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu3182125. Accessed June 12, 2022.- Ministério da Saúde (Brasil), Protocolo Clínico e Diretrizes Terapêuticas da Doença Falciforme,
Feb. 19, 2018 - National Heart, Lung, and Blood Institute. Sickle Cell Disease. https://www.nhlbi.nih.gov/health-topics/sickle-cell-disease. Accessed February 23, 2022.
- Kato GJ, et al. Nat Rev Dis Primers. 2018;4:18010.
- Rees DC, et al.
Lancet . 2010;376(9757):2018-2031. - Kato GJ, et al. J Clin Invest. 2017;127(3):750-760.
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- Nader E, et al. Front Immunol. 2020 Mar 13;11:454.
- Kanter J, et al. Blood Rev. 2013 Nov;27(6):279-87.
Contact:
Steven Immergut (media)
+1 650-410-3258
simmergut@gbt.com
Courtney Roberts (investors)
+1 650-351-7881
croberts@gbt.com
Source:
2022 GlobeNewswire, Inc., source