A new drug for patients with spinal muscular atrophy (SMA) has just accelerated its testing process, according to a news report recently made by Biohaven Ltd.

“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,” said Karen Chen, PhD, chief executive officer of SMA Foundation.

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Taldefgrobep alfa is a recently developed drug that just received the Fast Track designation, a denomination granted by the United States Food and Drug Administration (FDA), the study team said. This event translates into a potentially faster process of reviewing this drug. With this designation, more patients with SMA could undergo treatment with taldefgrobep alfa, along with continuous communication with the FDA, they continued.

The Fast Track designation allows drugs that will potentially target unmet medical entities to get final approval in a faster manner. Importantly, the FDA had also previously granted taldefgrobep alfa the orphan drug designation for the treatment of SMA, the researchers noted.

This drug is a biologic investigational agent that targets myostatin by inhibiting its activity. This effect translates into better muscle function. Specifically, taldefgrobep alfa binds to lower myostatin levels while also performing functions as an Activin 2b receptor antagonist once the myostatin-taldefgrobep alfa complex has formed, the investigators noted. Taldefgrobep alfa is cataloged as an adnectin, a group of proteins based on human fibronectin with a therapeutic class, they added.

This news brings hope to patients suffering from SMA, the researchers noted. This debilitating genetic disorder that affects motor neurons and, subsequently, muscle mass and overall growth currently lacks a cure. Hence, a novel drug that could prevent the impaired muscle growth characteristic of this disease could greatly benefit these patients, they added.


Biohaven’s Taldefgrobep alfa receives FDA fast track designation for spinal muscular atrophy. Biohaven Ltd. Published online February 21, 2023.