Ultragenyx to Sell Rare Pediatric Disease Priority Review Voucher 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.

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 (RARE) agreed to sell a Rare Pediatric Disease Priority Review Voucher (PRV) for $210 million. The PRV was received after FDA approval of Genglycos, a treatment for glycogen storage disease type Ia (GSDIa).

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.