Aflibercept

Issues at a Catalent facility — now owned by Novo Nordisk — have resulted in a regulatory delay for another drug, this time for Scholar Rock's spinal muscular atrophy (SMA) treatment apitegromab. The FDA had been due to render a decision on the fully human monoclonal antibody by September 22, but has now issued a complete response letter (CRL). Disclosing the news on Tuesday, Scholar Rock said the rejection is related to previously disclosed observations at Catalent's Indiana fill-finish facility, which are not specific to apitegromab. The company noted that the CRL did not cite any other approvability concerns. Known issues Scholar Rock had highlighted the issues at the Catalent site during its second-quarter earnings announcement last month. "We are continuing to work closely with Catalent…on the FDA's manufacturing observations so that we can resubmit the apitegromab [biologics license application] as soon as possible," remarked chief executive David Hallal on Tuesday. Issues at Catalent's Indiana facility were also recently cited by Regeneron Pharmaceuticals after it received a CRL from the FDA for its experimental lymphoma drug odronextamab, as well as a three-month extension to filings for its high-dose version of Eylea (aflibercept). Reports have suggested that the agency uncovered repeated quality control failures at the Catalent site, including foreign particle contamination, bacterial risks and inadequate corrective actions, with at least 20 deviations logged since July 2024. While analysts had noted the possibility of Scholar Rock receiving a CRL for apitegromab, those at BMO Capital Markets said it was more likely that as the drug is produced in the same fill-finish facilities as high-dose Eylea, it would in all likelihood face "a possible minor…delay." Hopes for speedy resolution According to Scholar Rock, Catalent submitted a response to the FDA's observations in early August and has continued to work to take corrective action. Once the issues have been fixed, Scholar Rock will resubmit its application for apitegromab, hoping that the agency "will be able to act expeditiously" on the re-filing. Apitegromab, which inhibits myostatin activation by selectively binding the pro- and latent forms of myostatin in the skeletal muscle, is also under regulatory review in Europe, with a decision expected in the second half of 2026.

iStock, cagkansayin 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 .