FDA accepts NDA for voxelotor, an oral, once-daily therapy for the treatment of sickle cell disease

Global Blood Therapeutics, Inc. announced that the 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.

The FDA 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. 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 PDUFA, a Priority Review targets a review time of six months compared to a standard review time of 10 months. The NDA for voxelotor is supported by data from the multi-national Phase III HOPE Study of voxelotor in patients ages 12 and older with SCD who enrolled in the study from 60 institutions across 12 countries. The HOPE Study results were recently published in The New England Journal of Medicine. Voxelotor has been granted FDA Breakthrough Therapy.