FDA Approves Ultragenyx Gene Therapy for Sanfilippo Syndrome
RARE is trading near its 52-week low of $12.73 (14% above the low) on elevated volume (2.6× avg).
Summary
The FDA approved Ultragenyx's gene therapy UX111, also known as FAYUVI, for Sanfilippo syndrome type A, a rare and fatal pediatric neurodegenerative disorder. This is the company's second gene therapy approval in under a month, following GENGLYCOS for GSDIa in August. The approval validates Ultragenyx's gene therapy platform and opens a new commercial market, though the patient population is small. The FDA granted full approval, removing confirmatory requirements, and Ultragenyx set a U.S. price of $3.95 million per one-time IV AAV9 treatment; shipments to treatment centers have begun. This follows the recent failure of apazunersen in Angelman syndrome, making this approval a critical positive catalyst for the stock, which jumped on the news.
Updated with a Wiseek News report · What changed
Updates
· Wiseek News — The FDA granted full approval, removing confirmatory requirements, and Ultragenyx set a U.S. price of $3.95 million per one-time IV AAV9 treatment; shipments to treatment centers have begun.
At the time of this announcement, RARE was trading at $14.54 on NASDAQ in the Life Sciences sector, with a market capitalization of approximately $1.4B. The 52-week trading range was $12.73 to $39.89. This news item was assessed with positive market sentiment and an importance score of 9 out of 10. Source: Seeking Alpha.