Ultragenyx Pharmaceutical Inc. (RARE) Receives FDA CRL for UX111, Plans Quick Resubmission

Ultragenyx Pharmaceutical Inc. received a Complete Response Letter from the FDA for its Biologics License Application for UX111, a gene therapy for Sanfilippo syndrome type A. The FDA highlighted chemistry, manufacturing, and controls (CMC) issues but recognized the therapy's efficacy. The company plans to resolve the CMC concerns and resubmit the application, expecting a six-month review. Ultragenyx is advancing other promising therapies, including UX143 for osteogenesis imperfecta and GTX-102 for Angelman syndrome, which received the FDA Breakthrough Therapy designation.

Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) recently received a Complete Response Letter (CRL) from the U.S. Food and Drug Administration (FDA) regarding its Biologics License Application for UX111, a gene therapy for Sanfilippo syndrome type A. The FDA highlighted chemistry, manufacturing, and controls (CMC) issues but recognized the therapy's efficacy. The company plans to address these concerns and resubmit the application, expecting a six-month review period [1].

The FDA's feedback underscores the importance of manufacturing standards in gene therapy approvals. Ultragenyx has indicated that the issues are "readily addressable," suggesting a manageable delay in the approval process. The company's focus on UX111 is part of its broader strategy to advance gene therapies for rare diseases.

In addition to UX111, Ultragenyx is advancing other promising therapies. UX143, a treatment for osteogenesis imperfecta (OI), is currently in a pivotal Phase 3 study and is expected to provide final data by the end of 2025. The FDA Breakthrough Therapy designation for GTX-102, a gene therapy for Angelman syndrome, further highlights Ultragenyx's commitment to developing innovative treatments for rare diseases [2].

The company's recent announcements also include a shelf registration for up to US$95.94 million in common stock linked to an Employee Stock Ownership Plan (ESOP)-related offering. This move underscores Ultragenyx's commitment to funding its pipeline and ensuring financial flexibility as it addresses regulatory requirements [1].

Investors should monitor Ultragenyx's progress in resolving the CMC issues for UX111 and the ongoing trials for UX143 and GTX-102. The company's ability to navigate regulatory challenges and maintain clinical momentum will be critical for its future growth and potential profitability.

References:
[1] https://simplywall.st/stocks/us/pharmaceuticals-biotech/nasdaq-rare/ultragenyx-pharmaceutical/news/will-fda-response-and-new-stock-offering-change-ultragenyx-p
[2] https://seekingalpha.com/article/4805424-ultragenyx-july-selloff-creates-buying-opportunity-ahead-of-ux143-catalyst