Global Blood Therapeutics Inc. (NASDAQ: GBT) shares shot up on Monday after the company announced that it received an update from the U.S. Food and Drug Administration in regards to its sickle cell disease (SCD) treatment.
Essentially the agency informed GBT that it agrees with the company’s proposal relating to use of an accelerated approval pathway for voxelotor for the treatment of SCD. GBT plans to submit an NDA for voxelotor for the treatment of SCD under this pathway.
GBT proposed that by raising hemoglobin, voxelotor is reasonably likely to reduce strokes in SCD patients. As part of these discussions, the FDA agreed that transcranial Doppler flow velocity would be an acceptable primary endpoint in a post-approval confirmatory study to demonstrate stroke risk reduction.
The company plans to request a pre-NDA meeting for the first quarter of 2019 and intends to provide further details regarding its plans and timing for an NDA submission as well as additional specifics on the transcranial Doppler confirmatory study following this meeting.
Ted W. Love, M.D., president and CEO of GBT, commented:
Gaining U.S. regulatory alignment for voxelotor under the accelerated approval pathway is a significant achievement for SCD patients and their families. The FDA has been innovative in considering new endpoints and accelerated approvals to bring desperately needed new therapies to other serious rare diseases. The FDA’s openness to now apply this same approach to SCD is well founded based upon the compelling evidence that more severe hemolytic anemia is associated with higher morbidity and mortality. We commend the FDA, and the broader SCD community, for its continuous commitment toward understanding the critical need for new clinical endpoints that support the development of much needed new therapies to treat this devastating disease.
Shares of GBT were last seen up about 35% at $42.50, in a 52-week range of $30.15 to $68.05. The consensus analyst price target is $81.92.