Scholar Rock (NASDAQ:SRRK) said it has resubmitted its Biologics License Application (BLA) to the U.S. Food and Drug Administration for apitegromab as a treatment for children and adults with spinal muscular atrophy (SMA), positioning the company for a new review cycle and a potential regulatory decision by late September.
Chairman and CEO David Hallal said the resubmission addresses the sole approvability issue cited in the company’s complete response letter (CRL) on the prior priority review action date: findings from a routine inspection at the Catalent Indiana fill-finish facility, which is owned and operated by Novo Nordisk. Hallal said the updated filing includes both Catalent Indiana and an additional U.S.-based fill-finish facility, creating “two independent paths” to approval.
Resubmission includes two fill-finish facilities
Scholar Rock also added a second fill-finish facility to the resubmission following a March 3 Type C meeting with the FDA. Hallal said the company and the agency aligned to include the second facility based on accelerated timelines for commercial supply, and he characterized the two-site approach as providing “significant optionality.”
What the FDA review could look like
President of R&D Akshay Vaishnaw said the resubmission updates were “limited in scope” and largely consisted of a standard safety update from ongoing clinical studies and the early access program, along with draft labeling from the last round of FDA interactions.
Vaishnaw said Scholar Rock expects an FDA decision on whether the resubmission is accepted within 30 days, with “up to a six-month review period.” Hallal said the company expects the resubmission to be treated as a Class 2 resubmission because the Catalent reinspection has not yet occurred and the second facility requires new review data.
Vaishnaw outlined the expected steps for the Catalent Indiana path:
- An unannounced FDA reinspection “in the coming period.”
- Potential issuance of a Form 483 at the conclusion of the inspection; Vaishnaw noted this is typical and “would not necessarily limit” the FDA’s ability to favorably reclassify the site.
- Novo Nordisk would have 15 business days to respond to any observations, followed by FDA review and reclassification as appropriate.
With two facilities included, Vaishnaw said the FDA has guided Scholar Rock that whichever facility supports the most rapid approval could remain in the BLA, while the other could be removed and later added via a supplemental BLA after approval.
Management repeatedly emphasized that approval could occur before the end of the six-month window, but said the company is framing expectations as “up to six months” while allowing the FDA to complete its review.
Commercial readiness, supply, and payer engagement
Hallal said the company is “ready now” for a U.S. launch and has used the extended timeline to prepare. Chief Operating Officer Keith Woods said Scholar Rock has continued disease-state education, expanded its specialty pharmacy network, built a “10,000-nurse home infusion network,” and continued payer engagement across national and regional plans as well as Medicare and Medicaid. He also said the company has been “testing and retesting” its Scholar Rock Supports program to help patients transition from prescription to treatment after approval.
On supply, Hallal said Scholar Rock expects to have “plenty of commercial apitegromab” available for launch, including from the second fill-finish facility alone if necessary. He said commercial product from the second facility is expected to be ready for release “several months” before a late-September action date.
In payer discussions, Woods said Scholar Rock is aiming for coverage policies that are not strictly tied to phase III inclusion and exclusion criteria, but instead “give a broader sense of utilization for the community.” Hallal acknowledged potential early payer headwinds, citing common dynamics such as coding, policy development, and the breadth of any eventual label, but said the company believes payers understand the unmet need in SMA.
Pricing, cash outlook, and myostatin positioning
Asked about pricing, Hallal said it is “still a little premature” to provide ranges, but said the company is anchored by factors including SMA’s rarity, disease severity, and what he called compelling clinical data showing a “return or gain of motor function as opposed to a loss.” Woods said Scholar Rock believes the budget impact to any single payer or government should be limited due to the small patient population, and said the company looks forward to providing more specifics later.
On the potential impact of most-favored-nation (MFN) dynamics, Hallal said Scholar Rock’s global plans are unchanged and the company sees “very little impact” to its launch plans.
Chief Financial Officer Vikas Sinha said the company does not expect material incremental cash burn ahead of approval, noting that many expenses are headcount-related and that launch costs are “gated” by timing decisions but are not expected to be significant changes. He also said the company expects a priority review voucher (PRV) “to come on the approval,” and referenced “$150 million more” potentially available at approval from lenders.
Hallal also addressed questions about competition and the broader myostatin landscape, saying Scholar Rock believes it has demonstrated an ability to target myostatin “safely and effectively through phase III” and expects to pursue additional rare neuromuscular indications.
Europe: MAA review and dependency on FDA site clearance
For Europe, Hallal said Scholar Rock’s Marketing Authorization Application (MAA) is progressing well and the company expects a mid-2026 decision from the European Medicines Agency, with launch preparations underway and an anticipated start “beginning with Germany.” Vaishnaw reiterated that European approval requires FDA clearance of the Catalent Indiana facility, and said the EMA is aware of the site’s progress and “comfortable with the review timeline” that incorporates FDA clearance.
On whether a shift to the second fill-finish facility could affect the European timeline, Vaishnaw said the company is focused on a successful Catalent reinspection, adding that discussions with EMA’s CHMP have been constructive and that the company would address any needed changes if circumstances evolve.
About Scholar Rock (NASDAQ:SRRK)
Scholar Rock is a clinical-stage biotechnology company focused on the discovery and development of protein therapeutics that selectively target growth factors involved in disease processes. The company’s research platform is designed to modulate endogenous growth factor activation and signaling with high specificity, aiming to restore normal biological function across a range of disorders. Scholar Rock’s approach is distinguished by its emphasis on engineering antibodies and biologics that interact with growth factor precursors or latent complexes rather than the active form, potentially offering improved safety and efficacy profiles.
The company’s lead program, SRK-015 (appercept), is an investigational monoclonal antibody targeting the activation of latent myostatin proproteins and is being evaluated for the treatment of spinal muscular atrophy (SMA).
