Biohaven Ltd. announced that it received Fast Track designation from the U.S. Food and Drug Administration (FDA) for taldefgrobep alfa, a novel anti-myostatin adnectin, for the treatment of spinal muscular atrophy (SMA). Fast Track designation enables important new drugs to reach patients earlier by facilitating more frequent communications with the FDA and expeditious review of a drug which treats a serious condition and fills an unmet medical need. Biohaven previously received orphan drug designation from the FDA for taldefgrobep in the treatment of SMA.
SMA is a rare, progressively debilitating motor neuron disease in which development and growth of muscle mass are compromised, resulting in progressive weakness and muscle atrophy, reduced motor function, impaired quality of life and often death. Inhibition of myostatin, a naturally occurring protein that limits skeletal muscle growth, an important process in healthy muscular development, is a potential therapeutic target for SMA.
Taldefgrobep has the potential to be a novel therapy to be used in combination with disease modifying therapies to enhance muscle function by blocking myostatin activity. Taldefgrobep’s novelty in a field of myostatin inhibitors is based on its mechanism of action. It binds to myostatin to both lower overall myostatin levels and also function as a receptor antagonist, thereby blocking myostatin signaling in skeletal muscles.
Lindsey Lee Lair, M.D., M.B.A., F.A.A.N, Vice President, Clinical Development, Biohaven commented, “We are very pleased the FDA granted Fast Track designation for taldefgrobep alfa for the treatment of SMA. Children and adults living with SMA experience significant muscle weakness and functional impairments affecting their quality of life daily, and a substantial unmet medical need persists. We are excited about the potential for taldefgrobep alfa to improve the lives of patients and families affected by SMA.”
Karen Chen, Ph.D., CEO, SMA Foundation, added, “Fast Track designation from the FDA underscores the high unmet medical need in SMA and supports the need for additional novel combination therapies in children and adults living with this progressive neurologic disease. The SMA Foundation is heavily investing in supporting the development of additional muscle and neuromuscular regenerative medicine therapies that provide functional benefits to patients. We are happy to see the commitment of health authorities to rapidly advance the development of new therapies”
As a leader in innovative trials addressing neurodegenerative diseases, Biohaven is currently enrolling a Phase 3 clinical trial of taldefgrobep in SMA: A Study to Evaluate the Efficacy and Safety of Taldefgrobep Alfa in Participants with Spinal Muscular Atrophy (RESILIENT) (NCT05337553).