Scholar Rock (SRRK) Receives Fast Track Designation from U.S. FDA for Apitegromab for the Treatment of Patients with Spinal Muscular Atrophy

Scholar Rock (NASDAQ: SRRK), a clinical-stage biopharmaceutical company focused on the treatment of serious diseases in which protein growth factors play a fundamental role, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation for apitegromab, a selective inhibitor of myostatin activation, for the treatment of patients with Spinal Muscular Atrophy (SMA). Fast Track designation is intended to facilitate the development and expedite the review of drugs to treat serious conditions and get new drugs to patients earlier. Through Fast Track, Scholar Rock is eligible to submit a rolling Biologic License Application (BLA) for apitegromab if relevant criteria are met.

“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 Tony Kingsley, President and CEO of Scholar Rock. “We believe the recently announced top-line data from the TOPAZ Phase 2 trial showed the transformative potential of apitegromab for patients with SMA.”

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 Yung Chyung, M.D., Chief Medical Officer of Scholar Rock. “The majority of non-ambulatory patients in our TOPAZ trial experienced increases in Hammersmith scores, highlighting the therapeutic potential of apitegromab to address motor function impairments in this patient population.”

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