In its rejection letter, the FDA flagged problems at a third-party fill-finish site owned by Novo Nordisk. Issues at this site have previously been investigated by the regulator.
The FDA has declined to approve Scholar Rock’s investigational myostatin blocker apitegromab for spinal muscular atrophy, citing issues at a third-party manufacturer.
In particular, the regulator flagged “observations” during a routine inspection at Catalent Indiana LLC, a fill-finish site that was acquired by Novo Nordisk in December last year. “The observations are not specific to apitegromab,” Scholar Rock said in a news release on Tuesday, adding that the FDA “did not cite any other approvability concerns,” including problems with efficacy or safety.
Scholar Rock is working closely with Catalent Indiana regarding the FDA’s observations, building up to a resubmission “as soon as possible,” CEO David Hallal said on Tuesday. The biotech has not yet provided specific timing for its resubmission.
Tuesday’s rejection did not come as a surprise to BMO Capital Markets. In a note to investors, analysts said the FDA’s decision is “in line with our expectations,” nevertheless noting that the uncertain timing of Scholar Rock’s refiling “could create more downside” to the company’s shares. Shares of the biotech dropped 12.5% in pre-market trading Tuesday.
That the FDA did not ask for additional efficacy or safety data is a “mild positive” for Scholar Rock, BMO added, writing that it could “mitigate further concerns” surrounding the rejection.
To back its application for apitegromab, Scholar Rock filed data from the Phase III SAPPHIRE study, which in October 2024 demonstrated a significant improvement in motor function. Benefits were apparent as early as 8 weeks and further improved through 52 weeks of follow-up, the company said at the time.
Last month, STAT News made public a report of the FDA’s investigation at the same Catalent facility, documenting poor quality control and substandard equipment upkeep. The report flagged “atypical extrinsic particles” such as cat hair, as well as bacterial contaminations and pest infestations. The plant also “failed to demonstrate a root cause of the contamination” or make changes to prevent future issues.
The report also noted that these problems have been going on for years, with some cases reaching as far back as June 2022.
Novo told STAT at the time that it was taking these findings “seriously” and is taking “appropriate actions” to address the issues “promptly and holistically.”
Catalent Indiana’s facility tripped up Regeneron in its dealings with the FDA as well. Last month, the pharma announced that two decisions for its high-dose formulation of Eylea have been pushed into the fourth quarter, likewise because of problems at this Catalent facility. The FDA was originally set to deliver a decision for these applications on Aug. 19.
