Ultragenyx to Sell Priority Review Voucher for $210 Million; 20% Payable to NIH

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.

Ultragenyx Pharmaceutical (RARE) shares rose 3.2% after agreeing to sell a Rare Pediatric Disease Priority Review Voucher for $210M, awarded following FDA approval of GENGLYCOS for glycogen storage disease. The company plans to use proceeds to support rare disease therapy development and advance profitability, according to Ultragenyx.

Ultragenyx Pharmaceutical (RARE) sold a Rare Pediatric Disease Priority Review Voucher for $210M, according to the company. Proceeds will support rare-disease programs. RARE shares rose 4% at the time of writing, despite broader market declines. This is Ultragenyx's third PRV sale, with previous sales in 2018 totaling $210.6M.

Ultragenyx (RARE) agreed to sell a Rare Pediatric Disease Priority Review Voucher for $210M, received after FDA approval of GENGLYCOS. The deal, advised by Jefferies, aims to support the company's profitability path. The transaction is pending regulatory approval.

Ultragenyx Pharmaceutical (RARE) agreed to sell a Rare Pediatric Disease Priority Review Voucher for $210M. The voucher was received after FDA approval of GENGLYCOS, a treatment for glycogen storage disease type Ia. The deal, subject to closing conditions, will provide non-dilutive capital to advance rare disease therapies and support profitability. Jefferies LLC and Gibson Dunn are advising Ultragenyx.