Scholar Rock Holding Corp（SRRK）のニュースとイベントSRRKScholar Rock Holding Corp

Scholar Rock Removes Catalent Indiana as Apitegromab Commercial Fill-Finish Facility

Scholar Rock (SRRK) announced it has removed Catalent Indiana (NVO) as a commercial fill-finish facility from its apitegromab Biologics License Application for the treatment of spinal muscular atrophy in the U.S., under guidance from FDA. FDA review of the BLA is progressing well with the Company's alternate fill-finish facility, and Scholar Rock continues to expect an FDA approval decision by the September 30 Prescription Drug User Fee Act date. Scholar Rock also announced that, following the August 7 customer notification that FDA classified the Catalent Indiana April 2026 general site inspection as Official Action Indicated, the Company has withdrawn its apitegromab Marketing Authorisation Application via written procedure with the Committee for Medicinal Products for Human Use, which concluded on August 20. Scholar Rock will remove Catalent Indiana as a commercial fill-finish facility from the apitegromab MAA and resubmit the application with the alternate fill-finish facility to facilitate the most expeditious path to a CHMP opinion. In addition, Scholar Rock announced that it is now on track to submit a Japanese New Drug Application for apitegromab for the treatment of children and adults with SMA by year end 2026, following alignment with Japan's Pharmaceuticals and Medical Devices Agency that no additional clinical studies are required. This alignment is based on PMDA's assessment of the ability of apitegromab to meet the criteria as described in recent guidelines on core conditions for filing without Japanese clinical data. In Japan, clinical studies in the Japanese population are typically required to support a JNDA filing. Scholar Rock engaged with PMDA regarding recent guidelines allowing JNDA submissions for specific orphan and rare disease drugs without domestic Japanese clinical trial data. The core conditions include a pivotal trial successfully completed outside of Japan, impractical patient recruitment due to small target patient populations, and available scientific and clinical evidence strongly indicating favorable benefit-risk profile for Japanese patients. Based on these criteria, the PMDA has agreed that an apitegromab JNDA may be filed without clinical data in Japanese patients. Scholar Rock's alternate fill-finish facility for apitegromab is a U.S.-based facility producing numerous commercial products with a strong track record of regulatory compliance, including recent successful FDA and EMA inspections. Robust supply of apitegromab commercial vials from the alternate fill-finish facility is available to support the global launch of apitegromab following regulatory approvals.

Scholar Rock's Apitegromab BLA Progressing, Expected FDA Approval

Scholar Rock (SRRK) announced that the FDA review of the apitegromab biologics license application, or BLA, for the treatment of children and adults with spinal muscular atrophy, or SMA, is progressing with the company's second fill-finish facility. The BLA remains on track for potential FDA approval by the September 30 Prescription Drug User Free Act action date. Scholar Rock's apitegromab BLA submission in March included two fill-finish facilities, in alignment with FDA guidance. These facilities were Catalent Indiana, part of Novo Nordisk (NVO). and a second facility, which provided two independent paths to a potential FDA approval. The company received a customer notification from Catalent Indiana on August 7 that the FDA has classified the April site inspection of Catalent Indiana as official action indicated. Scholar Rock will continue to collaborate closely with the FDA and under their guidance, will remove Catalent Indiana from the apitegromab BLA. FDA review of the apitegromab BLA will progress solely with the second fill-finish facility. At the March Type C meeting, FDA and Scholar Rock agreed to the data package required for FDA review of the second fill-finish facility. That data package was submitted in advance of the agreed upon timeline, and Agency review of the data is progressing well. Robust supply from the second fill-finish facility is available, with vials from that facility on site at a third-party provider awaiting packaging and labeling for commercialization upon FDA approval. The second fill-finish facility for apitegromab is a U.S.-based facility producing numerous commercial products and it is in good standing with FDA and EMA. "We continue to be very pleased with our second fill-finish facility," said David Hallal, board chair and CEO of Scholar Rock. "The data package that we agreed upon with the FDA at our Type C meeting has been submitted, the FDA review of the facility is advancing, robust supply for commercialization is ready and awaiting packaging and labeling at a third-party site, and importantly, the facility has demonstrated a strong track record of compliance, including recent successful FDA and EMA inspections. As we work closely with the FDA on the final steps of the apitegromab BLA review process, we are ready to bring the world's first muscle-targeted treatment to children and adults with SMA."