Ultragenyx BLA for UX111 Accepted for FDA Review

Ultragenyx Pharmaceutical ( (RARE) ) has issued an update.

On April 2, 2026, Ultragenyx Pharmaceutical announced that the U.S. Food and Drug Administration accepted for review its resubmitted Biologics License Application for UX111, an AAV9 gene therapy for patients with Sanfilippo syndrome Type A, also known as MPS IIIA. The application seeks accelerated approval, and the FDA has set a Prescription Drug User Fee Act action date of September 19, 2026, marking a key regulatory milestone that could advance treatment options for this rare genetic disorder.

If successful, the UX111 review could strengthen Ultragenyx’s position in the rare disease gene therapy market by bringing a potential first-of-its-kind treatment closer to commercialization. The PDUFA timeline also provides greater visibility for patients, clinicians, and investors regarding when a regulatory decision on this therapy for MPS IIIA may be expected.

The most recent analyst rating on (RARE) stock is a Hold with a $21.00 price target. To see the full list of analyst forecasts on Ultragenyx Pharmaceutical stock, see the RARE Stock Forecast page.

Spark’s Take on RARE Stock

According to Spark, TipRanks’ AI Analyst, RARE is a Neutral.

The score is held down primarily by weak financial durability (large operating losses, substantial cash burn, and a sharply reduced equity base). Technicals also indicate a sustained downtrend. Offsetting these are earnings-call signals of a credible cost-reduction plan with profitability targeted for 2027 and supportive pipeline/regulatory milestones, but these benefits are still dependent on successful execution and regulatory outcomes.

To see Spark’s full report on RARE stock, click here.

More about Ultragenyx Pharmaceutical

Ultragenyx Pharmaceutical Inc. is a biopharmaceutical company focused on developing novel therapies for rare and ultrarare genetic diseases. The company’s pipeline includes gene therapies targeting serious inherited conditions with limited or no approved treatment options, aiming to address high unmet medical need in specialized patient populations.

Average Trading Volume: 2,440,113

Technical Sentiment Signal: Sell

Current Market Cap: $2.07B

For a thorough assessment of RARE stock, go to TipRanks’ Stock Analysis page.