Scholar Rock Prepares Apitegromab BLA Resubmission Following FDA Facility Reinspection

Scholar Rock is prepared to resubmit the biologics license application for apitegromab in spinal muscular atrophy (SMA) following a successful FDA reinspection of the Catalent Indiana fill-finish facility, with guidance for BLA resubmission and a U.S. launch following approval in 2026. The compliance status of the Catalent Indiana site remains the sole approvability issue cited in the company's complete response letter received last year.

The FDA issued a Complete Response Letter for the apitegromab BLA on September 23, 2025, related to observations identified during a routine general site inspection of Catalent Indiana LLC, a third-party fill-finish facility which was acquired by Novo Nordisk A/S in December 2024. The observations are not specific to apitegromab. The CRL did not cite any other approvability concerns, including apitegromab's efficacy and safety data or the third-party drug substance manufacturer.

Following a Type A meeting with the FDA in November that included Novo Nordisk and Cure SMA, the FDA issued a warning letter to the Catalent Indiana facility. Novo responded to the FDA by mid-December. The FDA then scheduled an early first-quarter meeting with Novo, and the agency had no additional requests to Novo's remediation plan at that meeting. The FDA sent a field team to Catalent Indiana after the meeting and again did not add requests to the remediation plan, instead stating its intent to conduct a site reinspection following routine manufacturing activities. Routine manufacturing activities resumed in late February.

On the resubmission trigger, the company would look for a successful re-inspection and some level of confidence that it was a successful re-inspection. Management noted it had reached an advanced stage with a draft label during the original review cycle and expects alignment on a final label relatively straightforward after resubmission, while cautioning that label details are ultimately up to the FDA.

Scholar Rock is also advancing a second fill-finish facility intended to add redundancy and support future commercial demand. Engineering runs are underway and additional manufacturing runs will follow, with expectations to file a supplemental BLA later this year. Additional manufacturing runs are planned for the second quarter, with expectations to submit a supplemental filing with the facility later in 2026.

Outside the U.S., the apitegromab marketing authorization application review continues with the European Medicines Agency, and the company expects a decision in mid-2026. European launch is anticipated in the second half of 2026, with Germany expected to be the first European market with patient access. European regulators are aware of developments around Catalent Indiana.

The company has built out critical commercial capabilities ahead of an anticipated launch. The SMA market continues to grow and now represents nearly $5 billion in global annual sales. The U.S. customer-facing team is active in the field, focused on disease education emphasizing SMA as a disease of the motor unit, consisting of both motor neurons and muscle. The company reported engagement across approximately 140 SMA treatment centers and 2,600 prescribing physicians and multidisciplinary care teams.

Patients receiving an SMN-targeted therapy through a specialty pharmacy will be able to access apitegromab through the same network.

Apitegromab is an investigational fully human monoclonal antibody inhibiting myostatin activation by selectively binding the pro- and latent forms of myostatin in the skeletal muscle. It is the first muscle-targeted treatment candidate in spinal muscular atrophy to demonstrate clinical success in a pivotal Phase 3 clinical trial. The FDA has granted Fast Track, Orphan Drug and Rare Pediatric Disease designations, and the EMA has granted Priority Medicines (PRIME) and Orphan Medicinal Product designations, to apitegromab for the treatment of SMA.

Redmile Group disclosed a buy of 1,316,390 shares of Scholar Rock Holding Corporation in a February 17, 2026, SEC filing, with the estimated transaction value at $49.37 million based on quarterly average pricing. The quarter-end value of the position increased by $84.58 million, which reflects both the additional shares acquired and the impact of market price movements. The post-trade stake totaled 5,220,846 shares valued at $229.98 million, representing 16.94% of the fund's 13F reportable assets under management.

As of February 17, 2026, shares of Scholar Rock Holding Corporation were priced at $46.45, up 25.7% over the past year. The company has approximately $365 million in cash as of December 31, with runway projected into 2027.

The Catalent Indiana fill-finish facility located in Bloomington, Indiana is currently owned and operated by Novo Nordisk. This facility is no longer part of Catalent, Inc., and Catalent, Inc. does not operate the facility.