Allison Bratzel

Gary Nachman

Kripa Devarakonda

Marc Frahm

Michael Yee

Tessa Romero

David Nierengarten

Andres Maldonado

Dennis Kennedy

Eric Schmidt

Etzer Darout

Evan Seigerman

Malcolm Hoffman

Srikripa Devarakonda

Thomas Deal

- The **U.S. Food and Drug Administration (FDA)** issued a **Complete Response Letter (CRL)** to Scholar Rock Holding Corp for its biologics license application (BLA) of **apitegromab**, a treatment for spinal muscular atrophy (SMA).  **[1727196_eb62316313644101b98dc6ff58eb9d74]** 
- The CRL was due to findings from a routine inspection at a third-party manufacturing facility, **Catalent Indiana LLC**, but **did not raise any concerns regarding the safety or efficacy of apitegromab itself**.  **[1727196_eb62316313644101b98dc6ff58eb9d74]** 
- Scholar Rock is working with **Catalent Indiana** to address the manufacturing observations and plans to **resubmit the BLA** once these issues are resolved; **Catalent Indiana** submitted a comprehensive response to the FDA in **early August 2025**.  **[1727196_eb62316313644101b98dc6ff58eb9d74]** 
- Despite the setback, analysts remain cautiously optimistic about Scholar Rock's future, citing positive progress in its SMA program and the drug's transformative potential.  **[1727196_eb62316313644101b98dc6ff58eb9d74]**

Scholar Rock Receives FDA Complete Response Letter for Apitegromab Due to Manufacturing Issues

- Scholar Rock Holding Corporation (SRRK) announced on **September 23, 2025**, that the **FDA** issued a **Complete Response Letter (CRL)** for its Biologics License Application (BLA) for **apitegromab**, intended for the treatment of spinal muscular atrophy (SMA).  **[1727196_0001104659-25-092317_tm2526733d1_8k.htm:1]** **[1727196_0001104659-25-092317_tm2526733d1_ex99-1.htm:1]** **[1727196_0001104659-25-092317_tm2526733d1_8k.htm:2]** 
- The CRL was solely related to **observations identified at Catalent Indiana LLC**, a third-party fill-finish facility, and these observations are **not specific to apitegromab**.  **[1727196_0001104659-25-092317_tm2526733d1_ex99-1.htm:0]** **[1727196_0001104659-25-092317_tm2526733d1_ex99-1.htm:1]** 
- The FDA's CRL **did not cite any other approvability concerns**, including apitegromab's efficacy and safety data or the third-party drug substance manufacturer.  **[1727196_0001104659-25-092317_tm2526733d1_ex99-1.htm:1]** 
- Scholar Rock intends to **resubmit the apitegromab BLA** once the manufacturing issues at Catalent Indiana LLC are resolved.  **[1727196_0001104659-25-092317_tm2526733d1_ex99-1.htm:1]**

Scholar Rock receives FDA Complete Response Letter for Apitegromab BLA

Scholar Rock Holding (SRRK)