Paediatric priority review vouchers are selling for more than $200 million
Two sales by Ultragenyx in a single week and a run of new FDA awards show how much the rare pediatric disease voucher is worth since Congress revived the programme in February.
In brief
Ultragenyx sold two rare pediatric disease vouchers for $210m and $203m in October, as the FDA published new awards for Isembyld and Atebrioz.
- Ultragenyx agreed to sell vouchers for $210 million and $203 million within a week in October 2026.
- The FDA published voucher notices for Scholar Rock’s Isembyld on 26 October and Mirum’s Atebrioz on 28 October.
- The programme lapsed in December 2024 and was revived in February 2026, with funding through September 2029.
The market for rare pediatric disease priority review vouchers has had a busy October. Ultragenyx agreed to sell two vouchers in a single week this month, for $210 million and $203 million, and the FDA has published formal notice of two further awards in the past few days.
The vouchers are granted when a drug for a serious rare disease that primarily affects children is approved. They entitle the holder to priority review of a later application, and they can be sold, which is why they matter so much to the small companies that win them.
Two sales in a week
Ultragenyx’s first sale, announced on 7 October 2026, came from the approval of Genglycos (pariglasgene brecaparvovec), its gene therapy for glycogen storage disease type Ia, and fetched $210 million. The second, announced on 14 October, came from Fayuvi (rebisufligene etisparvovec), the first FDA-approved treatment for Sanfilippo syndrome type A, and fetched $203 million. Neither buyer was named.
“The sale of this PRV delivers meaningful non-dilutive funding,” said Howard Horn, Ultragenyx’s chief financial officer, adding that it supports the company’s aim of reaching profitability. The company noted that Fayuvi itself had benefited from the proceeds of an earlier voucher sale, a reminder that the scheme can recycle capital within rare disease portfolios.
New awards keep coming
On 26 October the FDA published notice in the Federal Register that Scholar Rock had received a voucher for Isembyld (apitegromab-mstn), approved on 11 September 2026 for spinal muscular atrophy in patients aged two and over who are already on an SMN2-targeted therapy. A notice for Mirum’s Atebrioz (zilurgisertib), approved in September for fibrodysplasia ossificans progressiva, followed on 28 October.
The pipeline of potential awards continues. Rafael Holdings, which filed remibetadex for Niemann-Pick disease type C on 29 October, noted that approval would make the drug eligible for a voucher.
A voucher’s value depends as much on Congress as on the drug that earned it.
A programme with a deadline
These sales come from a programme that came close to disappearing. Its authorisation expired on 20 December 2024, and it was revived only when the Consolidated Appropriations Act of 2026, signed on 3 February, reinstated it through the Mikaela Naylon Give Kids a Chance Act. Funding now runs to September 2029. At the time of the revival, Fierce Pharma cited figures showing 63 vouchers awarded across 47 rare diseases since the programme began in 2012.
For sponsors, the lesson is mixed. A voucher can now be worth a nine-figure sum that materially changes the financing of a paediatric programme. But its value depends on Congress, and the next sunset is less than three years away.
