Earlier today clinical-stage biopharmaceutical firm Global Blood Therapeutics Inc. (GBT:NASDAQ) announced that the U.S. Food and Drug Administration (FDA) has accepted for filing the company's New Drug Application (NDA) seeking accelerated approval for voxelotor, an oral, once-daily therapy in development for the treatment of sickle cell disease (SCD). If approved, voxelotor would be the first therapy available to patients that targets hemoglobin polymerization, the root cause of SCD damage.
This timely announcement comes as September is designated as National Sickle Cell Awareness month. The company states that it is proud to show its support for the approximately 100,000 Americans affected by SCD. Together with the Sickle Cell Disease Association of America Inc., Global Blood Therapeutics will be hosting the 8th Annual Sickle Cell Disease Therapeutics Conference on September 10, 2019, in Washington, D.C.
In today's release, the firm noted that the FDA has granted Priority Review for the NDA for voxelotor, which provides for a six-month review, and assigned a Prescription Drug User Fee Act (PDUFA) target action date of February 26, 2020. The agency also indicated in the NDA filing acceptance notification letter that it is not currently planning to hold an advisory committee meeting to discuss the application for voxelotor.
Ted W. Love, M.D., the company's president and CEO, stated, "The FDA's acceptance of our NDA for voxelotor under Priority Review is a major milestone in the development of this investigational therapy and further illustrates the significance the Agency places on getting important and innovative treatments to individuals living with SCD as quickly as possible...We look forward to working with the FDA during this process, with the goal of potentially changing the treatment paradigm for SCD."
The company advised that Priority Review is granted to therapies that the FDA determines have the potential to provide a significant improvement in the safety or effectiveness of the treatment, diagnosis or prevention of a serious condition. Under the PDUFA, a Priority Review targets an accelerated review time of six months compared to a standard review time of 10 months.
The firm states that the NDA for voxelotor is supported by data from the multi-national Phase 3 HOPE Study of voxelotor in patients ages 12 and older with SCD who enrolled in the study from 60 institutions across 12 countries. The company indicated that the HOPE Study results were recently published in The New England Journal of Medicine, and that voxelotor has been granted FDA Breakthrough Therapy, Fast Track, Orphan Drug and Rare Pediatric Disease designations for the treatment of patients with SCD.
The company explains that Sickle Cell Disease (SCD) is a lifelong inherited blood disorder caused by a genetic mutation in the beta-chain of hemoglobin, which leads to the formation of abnormal hemoglobin known as sickle hemoglobin (HbS). Beginning in childhood, SCD patients typically suffer unpredictable and recurrent episodes or crises of severe pain due to blocked blood flow to organs, which often lead to psychosocial and physical disabilities.
Global Blood Therapeutics is based in South San Francisco, Calif., and describes its business as a clinical-stage biopharmaceutical company determined to discover, develop and deliver innovative treatments that provide hope to underserved patient communities. The firm is also developing another therapy for the potential treatment of sickle cell disease.
GBT shares opened higher today at $54.45 (+$6.96, +14.66%) over yesterday's close of $47.49. The stock has traded today between $52.61 and $56.50/share and is currently trading at $54.42 (+$6.93, +14.59%).[NLINSERT]
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