Posted by Michael Wonder on 05 Sep 2019
GBT announces U.S. FDA acceptance of new drug application and priority review for voxelotor for the treatment of sickle cell disease
5 September 2019 - NDA supported by data from Phase 3 HOPE study, which demonstrated statistically significant and sustained improvements in haemoglobin with voxelotor.
Global Blood Therapeutics today announced that the U.S. FDA has accepted for filing the company’s new drug application 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 target action date of 26 February 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.
