Palovarotene

The end of the FDA's rare pediatric PRV program could lead to increased value of the tropical disease PRVs, lawyers at Hyman, Phelps & McNamara recently suggested. The FDA approval of Vimkunya has given the chikungunya vaccine’s owner, Bavarian Nordic, an extra financial windfall.Bavarian Nordic has reached an agreement to sell a priority review voucher (PRV) gained from Vimkunya’s approval to an unnamed buyer for $160 million, the Danish company said Wednesday. The FDA awarded the PRV under its tropical disease program.Bavarian got the shot in 2023 as part of the company’s acquisition of Emergent BioSolutions’ travel health business for $270 million upfront. As the vaccine was originally licensed from the National Institutes of Health, the government research agency will receive 20% of the proceeds from the PRV sale.Gains from the PRV sale will be recorded as operating income, so Bavarian expects an increase in its projected profits for 2025.PRVs are coveted commodities in the biopharma industry because they can slash the FDA’s review time of drug applications from the standard 10 months to six months. And the price of PRVs seems to be on the rise.It was only in February 2024 that Valneva monetized a PRV obtained from the FDA approval of the U.S.’s first approved chikungunya vaccine, Ixchiq, for $103 million. The general understanding at that time was that PRVs could sell for about $100 million.A notable uptick in PRV prices started later in 2024. In August, Ipsen said it would receive $158 million from selling its PRV tied to the FDA approval of the company’s rare disease drug Sohonos.In three transactions unveiled in November and February, PTC Therapeutics, Acadia Pharmaceuticals and Zevra Therapeutics all sold their PRVs for $150 million.The rise in the monetary value of PRVs came as concerns mounted over declining supplies. The future of the FDA’s rare pediatric disease PRV program, which was designed to incentivize the development of therapies for rare pediatric diseases, came into serious question as the program was sunset in December 2024 after a lapse in its Congressional reauthorization. As a result, the FDA may only award PRVs under this pathway for drugs that have already received a rare pediatric disease designation before Dec. 20, 2024, and the agency may not award any such PRVs after Sep. 30, 2026.There have been calls for the renewal of the program, including at an FDA cell and gene therapy roundtable earlier this month. Still, there’s no clear sign that the initiative will be revived, and critics have raised concerns that the ability to sell PRVs at high prices distorted the original purpose of the program.The impact of the loss of the rare pediatric disease PRV program on the market supply of PRVs is significant. About 70% of all PRVs that have been awarded since 2009 were for therapies against rare pediatric diseases, according to a recent analysis by the law firm Hyman, Phelps & McNamara.In an article published in February, lawyers at HPM suggested the end of the rare pediatric PRV program could lead to increased value of the tropical disease PRVs, potentially to a level that’s on par with the record $350 million that AbbVie paid United Therapeutics in 2015.Meanwhile, the FDA just introduced a new PRV program, which is designed to shorten the drug review time to as little as one or two months. Called the “Commissioner’s National Priority Voucher” (CNPV), the coupon will be given to “companies aligned with U.S. national priorities,” the FDA said Tuesday. But unlike existing PRVs, CNPVs are non-transferrable, according to the FDA. As for Bavarian Nordic, the vaccine specialist is differentiating itself from rival Valneva not only when it comes to PRV sale prices. In May, the FDA and the CDC recommended a pause in the use of Valneva’s Ixchiq in people 60 years of age and older following postmarketing reports of some serious adverse events, including two deaths, in those who had received the vaccine.Before that, Bavarian’s Vimkunya in April picked up a recommendation from the CDC’s Advisory Committee on Immunization Practices for individuals aged 12 and older traveling to places where there is a chikungunya outbreak, as well as for people who plan extended stays in areas at elevated risk and for certain lab workers.Vimkunya was approved in April with a broader FDA label reaching individuals as young as 12 years of age, whereas Ixchiq is only cleared in adults. Ixchiq is a live-attenuated vaccine, compared with Vimkunya’s virus-like particle construct. 

Ipsen Pharmaceuticals sold its priority review voucher to an unidentified “large global pharmaceutical company” for $158 million, it said this week, the highest publicly announced price for one of the vouchers in about eight years. Ipsen obtained the rare pediatric disease voucher in August 2023 thanks to the approval of Sohonos (palovarotene), which is intended to help adults and children with the rare disease known as fibrodysplasia ossificans progressiva. Priority review vouchers, or PRVs, can be acquired through the approval of rare pediatric disease or rare tropical disease drugs, and then can be sold or used by companies to speed the FDA’s reviews of any new drug. The $158 million sale is about $50 million more than any of the other PRV sales since last August, which includes sales of between $100 million and $108 million for Krystal Biotech, Valneva, X4 Pharmaceuticals and Day One Biopharmaceuticals. The last time a PRV sold for more than $158 million was in 2016, when PaxVax Bermuda sold its PRV, acquired via the approval of its cholera vaccine Vaxchora, for $290 million to an undisclosed buyer. PRVs have typically sold for about $100 million since the voucher programs began more than a decade ago, with higher prices of more than $150 million only coming between 2014 and 2016. “The sale of the PRV generates further firepower for our ongoing medicine launches and external-innovation strategy, which has seen multiple additions to our portfolio at every stage of development in 2024,” Philippe Lopes-Fernandes, chief business officer at Ipsen, said in a statement.