Ultragenyx's Fayuvi Approval Adds $200M Voucher and $3.95M Price Tag
RARE is trading near its 52-week low of $12.73 (14% above the low).
Summary
The FDA approved Ultragenyx's Fayuvi (UX111) for Sanfilippo syndrome type A, the first-ever treatment for this fatal pediatric disorder. The company received a Priority Review Voucher worth roughly $200 million, a significant balance sheet boost. Ultragenyx set the wholesale acquisition cost at $3.95 million, and analyst Sami Corwin models $325 million in peak sales. William Blair rates the stock Market Perform, citing a lack of key value inflection points in the next 12 months. Management highlighted over 75% prescriber overlap with existing products and 300-400 identified US patients, supporting a focused commercial launch. This follows the August approval of Genglycos for GSDIa, marking Ultragenyx's second gene therapy approval in two months.
At the time of this announcement, RARE was trading at $14.45 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 7 out of 10. Source: Benzinga.