Ultragenyx to sell rare PRV for $210 million

Ultragenyx sold its Rare Pediatric Disease Priority Review Voucher for $210M, earned for Genglycos. Viatris agreed to acquire Pacira BioSciences for $1.65B, adding non-opioid pain therapies. Peter Young notes improving financing and M&A activity in biopharma despite policy and patent challenges.

Ultragenyx Pharmaceutical agreed to sell a rare pediatric disease priority review voucher for $210M, according to the company. The voucher was earned after FDA approval of Genglycos, a treatment for glycogen storage disease type Ia. The deal is subject to regulatory clearance and will provide non-dilutive capital for Ultragenyx's rare disease programs.

Ultragenyx Pharmaceutical will sell a Rare Pediatric Disease Priority Review Voucher for $210 million. The voucher was earned after FDA approval of Genglycos, a treatment for glycogen storage disease type Ia. The sale will provide non-dilutive capital to advance Ultragenyx's pipeline and move toward profitability, continuing a cycle of voucher monetization for development funding.

H.C. Wainwright reiterated a Buy rating and $25 price target on Ultragenyx (RARE) after its sale of a priority review voucher for $210M. The company's stock is down 52% over the past year. Ultragenyx also received FDA approval for FAYUVI, its gene therapy for Sanfilippo syndrome Type A, marking its second gene therapy approval.

Ultragenyx Pharmaceutical (NASDAQ: RARE) agreed to sell a rare pediatric disease priority review voucher for $210M, nearly half its cash reserves. The deal, subject to regulatory approval, follows the FDA's August approval of its Genglycos gene therapy. Shares rose 2.97% on the news. Ultragenyx plans to use proceeds to advance rare disease therapies and support profitability. The price is within the range of recent voucher transactions, such as Rocket Pharmaceuticals' $180M deal.

Ultragenyx Pharmaceutical (RARE) announced that the European Medicines Agency (EMA) has validated its Marketing Authorisation Application for rebisufligene etisparvovec, a gene therapy for Sanfilippo syndrome Type A. This validation confirms the filing is complete for review but does not guarantee approval. The company reported a US$115 million loss in Q2 2025 and has a cash runway of less than one year.