Muscle Targeting SMA Drug Enters FDA Reassessment...September Approval Milestone

Scholar Rock Holding Corp. (NASDAQ: SRRK) has resubmitted its Biologics License Application (BLA) to the U.S. Food and Drug Administration for apitegromab, a muscle-targeting monoclonal antibody for treating spinal muscular atrophy (SMA). The resubmission adds a second U.S. fill-finish facility alongside the Catalent Indiana site, operated under Novo Nordisk, which had previously been cited for quality concerns.

The FDA will decide within 30 days whether to accept the application and, if accepted, will complete its review within six months. Scholar Rock anticipates a final decision by late September 2026. Separately, the European Medicines Agency’s approval process for the same product is expected to conclude by mid-2026.

In its fourth-quarter and full-year 2025 earnings release, the company reported securing up to $550 million in new financing from Blue Owl Capital—approximately KRW 700 billion—to support the U.S. and European launches of apitegromab and advance its pipeline programs in SMA, facioscapulohumeral muscular dystrophy (FSHD) and SRK-439. As of year-end 2025, Scholar Rock held about $370 million (roughly KRW 500 billion) in cash, cash equivalents and short-term financial instruments, while continuing to report a net loss.

Recent SEC filings show that on March 13 the company granted new incentive stock awards to employees and executives under Nasdaq listing rules. On March 27, a Schedule 13G/A was filed to report a passive change in ownership by a major shareholder, reflecting ongoing disclosures related to staffing and share-ownership structure.

For the full year 2025, Scholar Rock recorded a net loss of approximately $380 million (late KRW 500 billion range). The company plans to utilize its new loan facilities to drive apitegromab’s commercialization and to advance its key clinical programs over the medium to long term.

Headquartered in Cambridge, Massachusetts, Scholar Rock is a late-stage biotech company leveraging a TGF-β family protein and myostatin biology platform to develop muscle-targeting monoclonal antibodies for rare neuromuscular diseases such as SMA.

Spinal muscular atrophy is a rare genetic neuromuscular disorder. As demand grows for complementary treatments that directly improve muscle function beyond SMN-targeting therapies, muscle-targeting antibodies like apitegromab and gene therapies have become major growth drivers in the global pharmaceutical and biotech sectors.