Ultragenyx prices Sanfilippo gene therapy at $4 million as it gains a second FDA approval; Bristol Myers halts Orum cancer drug

The FDA approved Fayuvi, the first gene therapy for Sanfilippo syndrome Type A, with a list price of $3.95 million.

This marks Ultragenyx's second gene‑therapy approval since August, earning sellable drug review vouchers that analysts say will strengthen the company’s balance sheet; the stock jumped double digits after the news.

Ultragenyx also warned of upcoming cost‑cutting measures following the failure of an Angelman syndrome candidate that had higher sales potential.

In a separate move, Bristol Myers Squibb said it is stopping development of an experimental cancer drug licensed from Orum Therapeutics, a program it funded with a $100 million upfront payment.

The announcements highlight the high price points of rare‑disease therapies and the strategic pipeline adjustments companies are making.