- BioMarin Pharmaceutical Inc. announced Monday morning that it has sold its latest Rare Pediatric Disease Priority Review Voucher (PRV) for $125 million.
- The company received the PRV from the Food and Drug Administration in April for the approval of Brineura, a treatment for the ultra-rare, fatal form of Batten disease.
- The biotech said the sales will be associated with a $25 million income tax expense and will reduce its GAAP Net Loss guidance to a loss of between $10 million to $30 million.
Priority Review Vouchers are part of an incentive program devised by the FDA to help encourage development of drugs that treat rare pediatric or tropical diseases. The vouchers can be used to expedite the regulatory process of another drug in development, and can be bought and sold to use at a later date.
Along with PRVs that are given upon approval, companies that develop these drugs also get extended commercial exclusivity, as well as tax incentives and cost breaks on R&D.
BioMarin did not disclose what company was willing to fork over the $125 million for the voucher. But this is the second PRV that the company has capitalized on. BioMarin received a PRV in 2014 for the approval of Vimizim (elosulfase alfa) that it sold later that year to Regeneron Pharmaceuticals Inc. for $67.5 million. The PRV was later used successfully by Regeneron and partner Sanofi SA to speed up the approval of PCSK9 inhibitor Praluent (alirocumab).
The FDA has awarded less than 20 PRVs since beginning the program in 2007. More than half of those vouchers have not yet been used, but several have been sold for extremely high price tags. The highest disclosed sale was to AbbVie Inc. from United Therapeutics for $350 million in 2015. AbbVie has yet to use the voucher.
The use of vouchers doesn’t always work out in drugmakers' favor, though. Sanofi paid $245 million in 2015 to Retrophin for its voucher; the French pharma later used the PRV to accelerate the approval process for its type 2 diabetes drug Soliqua — a combo of insulin Lantus and the GLP-1 agonist lixisenatide — in an effort to beat Novo Nordisk A/S to market with a similar combo. Sanofi’s efforts were for naught, however; both Soliqua and Novo’s Xultophy were approved by the FDA on the same day.
The most recent PRV was awarded to UltraGenyx Pharmaceutical Inc. earlier this month for the approval of Mepsevii.