Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) shares are trending on Friday.
First-Ever Treatment For Childhood Disease
Shares of the California-based biopharmaceutical company surged 2.55% to $14.87 in after-hours trading on Thursday following the Food and Drug Administration’s full approval of FAYUVI (rebisufligene etisparvovec-hopf), the first-ever treatment for Sanfilippo syndrome Type A.
FAYUVI, also known as UX111, is a single-dose AAV9 gene therapy designed for children with mucopolysaccharidosis type IIIA (MPS IIIA). It is an ultra-rare and fatal lysosomal storage disease that causes progressive neurodegeneration, with a median life expectancy of 15 years.
The approval marks Ultragenyx’s second gene therapy success and includes a Priority Review Voucher. The FDA incentive can be used to speed up the review of a future drug or sold to another drugmaker. Past vouchers have sold for hundreds of millions of dollars.
Emil D. Kakkis, chief executive officer and president of Ultragenyx, said the approval reflects “years of research from scientists and developers” and support from patient families, calling it a “historic milestone” for a community that had waited for a treatment option.
Trial Data Backs Long-Term Efficacy
The approval rests on data from the pivotal Transpher A trial and long-term follow-up, with clinical data now extending to nearly eight years. FAYUVI-treated patients (mITT population, N=17) showed a 23.5-point higher Bayley-III cognitive score (p<0.0001) than an external, untreated natural-history cohort (N=27).
FAYUVI is manufactured entirely in the U.S., at facilities in Bedford, Massachusetts and Columbus, Ohio. Ultragenyx expects the commercial product to reach Qualified Treatment Centers within 30 to 60 days.
Trading Metrics, Technical Analysis
Ultragenyx has a market capitalization of $1.43 billion, a 52-week high of $39.89 and a 52-week low of $12.73.
The Relative Strength Index (RSI) of RARE stands at 28.06.
The stock has fallen 50.19% over the past 12 months.
RARE is currently trading at about 6.52% of its 52-week price range.
Price Action: The biotech stock closed the regular session on Thursday at $14.50, up 12.58%, according to Benzinga Pro data.
Benzinga’s Edge Stock Rankings indicates Ultragenyx Pharmaceutical stock has a negative price trend across all time frames.
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Disclaimer: This content was partially produced with the help of AI tools and was reviewed and published by Benzinga editors.
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