Myostatin-Targeting Biologic Fast Tracked for Spinal Muscular Atrophy

The Company is currently evaluating the efficacy and safety of taldefgrobep alfa in patients with SMA in the phase 3 RESILIENT study.

The Food and Drug Administration (FDA) has granted Fast Track designation to taldefgrobep alfa for the treatment of spinal muscular atrophy (SMA).

Taldefgrobep alfa is a fully human anti-myostatin recombinant protein designed to lower free myostatin, a naturally occurring protein that limits skeletal muscle growth, and block myostatin signaling in skeletal muscles. The investigational therapy is currently being evaluated in the phase 3 RESILIENT study ( Identifier: NCT05337553). 

The study is enrolling ambulatory and nonambulatory patients 4 to 21 years old with SMA who are being treated with an SMA disease-modifying therapy and will remain on that same treatment regimen and dose throughout the trial. It is expected that taldefgrobep alfa, in combination with treatments that increase SMN protein (eg, nusinersen, risdiplam, onasemnogene abeparvovec), will provide further clinical improvement in patients with SMA.

“We are very pleased the FDA granted Fast Track designation for taldefgrobep alfa for the treatment of SMA,” said Lindsey Lee Lair, MD, MBA, FAAN, Vice President, Clinical Development, Biohaven. “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.”


Biohaven’s taldefgrobep alfa receives FDA Fast Track designation for spinal muscular atrophy. News release. Biohaven. Accessed February 21, 2023.