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FDA agrees accelerated approval pathway for GBT’s voxelotor


GBT plans to submit a new drug application (NDA) under this accelerated approval pathway following a meeting in early 2019.

Under the terms of a subpart H pathway, voxelotor will be subject to post-marketing studies to confirm its clinically meaningful benefit to SCD patients. GBT president and CEO Ted W Love said: “Gaining US regulatory alignment for voxelotor under the accelerated approval pathway is a significant achievement for SCD patients and their families.”

“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.”

Voxelotor is an oral, once-daily therapy. Professor Kim Smith-Whitley, clinical director and head of paediatrics at the Perelman School of Medicine’s Division of Haemtology, said: “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.