Scholar Rock Receives Fast Track Designation from the U.S. FDA for Apitegromab for the Treatment of Patients with Spinal Muscular Atrophy
- Builds on Priority Medicines (PRIME) designation recently granted by the
“We are delighted to receive Fast Track designation and look forward to working closely with the FDA towards our aim of establishing apitegromab as the potential first muscle-directed therapy for patients with SMA,” said
Fast Track is intended to fill an unmet medical need defined as providing a therapy where none exists or improving upon available therapies and is based on whether a drug will impact factors such as survival, day-to-day functioning, or if left untreated, progression to a more serious condition. In addition to Fast Track designation, apitegromab had previously received Orphan Drug and Rare Pediatric Disease designations from the FDA and PRIME and Orphan Medicinal Product designations from the EMA for the treatment of SMA.
“Recent Fast Track and PRIME designations granted by the regulatory agencies underscore the continuing unmet medical needs of patients with SMA,” said
Apitegromab is a selective inhibitor of the activation of myostatin and is an investigational product candidate for the treatment of patients with spinal muscular atrophy (SMA). Myostatin, a member of the TGFβ superfamily of growth factors, is expressed primarily by skeletal muscle cells, and the absence of its gene is associated with an increase in muscle mass and strength in multiple animal species, including humans.
Spinal muscular atrophy (SMA) is a rare, and often fatal, genetic disorder that typically manifests in young children. An estimated 30,000 to 35,000 patients are afflicted with SMA in
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