FDA Accepts Ultragenyx's Resubmitted BLA for UX111 Gene Therapy, Sets September PDUFA Date
Summary
Ultragenyx announced that the FDA has accepted its resubmitted Biologics License Application for UX111 gene therapy for Sanfilippo syndrome Type A, setting a PDUFA action date of September 19, 2026.
Key Events
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BLA Resubmission Accepted
The FDA has accepted Ultragenyx's resubmitted Biologics License Application (BLA) for UX111 (rebisufligene etisparvovec) AAV9 gene therapy for Sanfilippo syndrome Type A (MPS IIIA).
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PDUFA Action Date Set
The FDA has set a Prescription Drug User Fee Act (PDUFA) action date of September 19, 2026, for the potential accelerated approval of UX111.
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Addresses Prior Regulatory Setback
This acceptance follows a previous Incomplete Response Letter for the UX111 BLA, indicating progress in addressing prior FDA concerns and moving the program forward.
Analysis
This filing provides a significant positive update on Ultragenyx's UX111 gene therapy program, which previously received an Incomplete Response Letter from the FDA. The acceptance of the resubmitted Biologics License Application (BLA) for accelerated approval for Sanfilippo syndrome Type A removes a key regulatory hurdle and establishes a clear timeline for a potential approval decision with the PDUFA date set for September 19, 2026. This development is crucial for the company, especially as its stock is trading near 52-week lows, and could provide a much-needed catalyst.
At the time of this filing, RARE was trading at $19.10 on NASDAQ in the Life Sciences sector, with a market capitalization of approximately $2.1B. The 52-week trading range was $18.29 to $42.37. This filing was assessed with positive market sentiment and an importance score of 9 out of 10.