Global Blood Therapeutics, Inc. (GBT) (Nasdaq: GBT) today announced that the U.S. Food and Drug Administration (FDA) has informed GBT through discussions and written correspondence that the agency agrees with the Company’s proposal relating to use of an accelerated approval pathway for voxelotor for the treatment of sickle cell disease (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 (TCD) flow velocity would be an acceptable primary endpoint in a post-approval confirmatory study to demonstrate stroke risk reduction.
GBT 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 TCD confirmatory study following this meeting.
“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,” said Ted W. Love, M.D., president and chief executive officer of GBT. “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.”
“Acute and chronic brain injury that frequently manifests as cognitive disorders and includes severe complications such as stroke has a major impact on a patient’s quality of life and future productivity. Our current approach to mitigating these risks via chronic transfusions has significant downsides and associated lifelong complications,” said Kim Smith-Whitley, M.D., clinical director of the Division of Hematology and professor of pediatrics at the Perelman School of Medicine at the University of Pennsylvania, and director of the Comprehensive Sickle Cell Center at the Children's Hospital of Philadelphia. “A once-daily, oral therapy that has the potential to safely improve the anemia of SCD and thereby preserve brain function would be a major breakthrough. I am encouraged by the FDA’s acknowledgement of the urgent need for new treatment options for the SCD community.”
“Despite the complex nature of the disease, SCD patients have had limited treatment options for decades, and it is critical that we continue to advance the development of new medicines that address not only the symptoms, but also the underlying cause and the chronic and cumulative damage to organs,” said Alexis Thompson, M.D., president of the American Society of Hematology. “The recent workshop we held in partnership with the FDA emphasized the need for new clinical endpoints beyond measuring pain and pain crisis and is an important step towards allowing us to better evaluate the impact of new innovative therapies for SCD.”