Neurological

Apitegromab Fast Tracked for spinal muscular atrophy

The Food and Drug Administration (FDA) has given apitegromab the fast-track designation for the treatment of patients with spinal muscular atrophy (SMA).

Apitegromab is a selective inhibitor of the inactive form of myostatin to block its activation in muscles. Myostatin is mainly expressed by skeletal muscle cells to inhibit muscle growth. The company believes that inhibiting myostatin will improve motor function in patients with SMA.

The name is supported by topline data from the 12-month phase 2 TOPAZ study (ClinicalTrials.gov: NCT03921528), which tested the efficacy and safety of apitegromab, given intravenously every 4 weeks, in 58 patients of age from 2 to 21 years of age was rated type 2 and type 3 SMA. The results showed that apitegromab showed clinical improvements in motor skills based on the revised Hammersmith scale and the expanded Hammersmith functional motor scale. The most common treatment-related adverse events were headache, pyrexia, upper respiratory tract infections, cough, and nasopharyngitis.

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“We are very pleased to be recognized as a Fast Track and look forward to working closely with the FDA to establish apitegromab as the potential first muscle-directed therapy for patients with SMA,” said Tony Kingsley, President and CEO of Scholar Rock. “We believe the recently announced top-line data from the TOPAZ Phase 2 trial demonstrated the transformative potential of apitegromab for patients with SMA.”

The FDA’s fast-track designation enables accelerated review of therapies for the treatment of serious or life-threatening conditions. In general, the name is given to therapies that are expected to affect factors such as survival and daily functioning.

References

  1. Scholar Rock receives Fast Track Award from the US FDA for apitegromab for the treatment of patients with spinal muscular atrophy. [press release]. Cambridge, MA: Scholar Rock; May 24, 2021.
  2. Scholar Rock announces positive 12-month top-line results from the TOPAZ phase 2 clinical trial evaluating apitegromab in patients with type 2 and type 3 spinal muscular atrophy (SMA). [press release]. Cambridge, MA: Scholar Rock; April 6, 2021.

This article originally appeared on MPR

Subjects:

Neuromuscular Disorders Treatments for Spinal Muscular Atrophy

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