RARE Daily

FDA Grants Fast Track Designation to Biohaven’s Taldefgrobep Alfa for Spinal Muscular Atrophy

February 22, 2023

Rare Daily Staff

The U.S. Food and Drug Administration granted Fast Track designation for Biohaven’s taldefgrobep alfa for the treatment of spinal muscular atrophy.

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 spinal muscular atrophy.

Spinal muscular atrophy (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. The underlying pathology of SMA is caused by insufficient production of the SMN (survival of motor neuron) protein, essential for the survival of motor neurons, and is encoded by two genes, SMN1 and SMN2. 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, a novel anti-myostatin adnectin, 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.

“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,” said Lindsey Lee Lair, vice president, Clinical Development at Biohaven.

Biohaven is currently enrolling a phase 3 clinical trial of taldefgrobep in SMA.

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