- Novartis AG will pay $130 million to acquire a Priority Review Voucher from Ultragenyx Pharmaceutical Inc. in a move likely aimed at accelerating regulatory review for one of several potential blockbuster medicines the Swiss pharma plans to file for approval sometime in 2018.
- Ultragenyx received the voucher earlier this year when it secured U.S. approval of its rare pediatric disease drug Mepsevii (vestronidase alfa). Akin to a regulatory "fast pass", the vouchers can be used to shorten review by the Food and Drug Administration of a New Drug Application to six months
- While Novartis has made no public mention of its plans, several experimental treatments set for 2018 submissions could be the intended beneficiary of accelerated consideration by the FDA.
Priority Review Vouchers (PRVs) are a sought-after asset, both by the biotechs that can sell them for hundreds of millions of dollars and by the pharmas that frequently buy the regulatory coupons to leapfrog competition.
Drug makers can obtain PRVs through two programs aimed at encouraging development of new treatments for rare pediatric diseases or under-studied tropical maladies. If a biotech or pharma secures U.S. approval of a treatment for one of the diseases covered by either program, the FDA awards a PRV to the application's sponsor.
PRVs can be used on any subsequent new drug filing, shortening review times from approximately 10 months to six, and can be traded or sold to any other company. While four months may not seem like much, it can be a crucial window of time to secure market share ahead of a competitor. And for some drugs expected to be blockbuster products, four months of sales can add up to a hefty sum.
Companies don't always disclose the sale or purchase of a PRV immediately, but nine transactions have been announced publicly. A tenth was disclosed in a second-quarter filing by Gilead, although no other details were revealed other than that it had been acquired in 2016.
Publicly disclosed PRV acquisitions
|Date sold or disclosed||Sold by||Bought by||Used for||Price paid, millions|
|Jul. 2014||BioMarin||Sanofi, Regeneron||Praluent||$67.5|
|Nov. 2014||Knight Therapeutics||Gilead||Odefsey||$125|
|Aug. 2015||United Therapeutics||AbbVie||N/A||$350|
|Jun. 2017||?||ViiV Healthcare (GSK)||Juluca||$130|
While some of the first PRVs were sold for eye-opening figures, prices now appear to have stabilized between $125 million and $130 million.
In addition to the PRV just acquired from Ultragenyx, Novartis already has an unused PRV in its possession from the August approval of its CAR-T therapy Kymriah (tisagenlecleucel) for pediatric acute lymphoblastic leukemia.
PRVs don't expire, so Novartis could choose to stockpile its PRVs for later use. But the Swiss pharma does plan to file in 2018 for U.S. approval of three experimental medicines pegged as future blockbusters: a multiple sclerosis medicine known as siponimod, the eye drug brolucizumab and a sickle cell disease drug called crizanlizumab.
Brolucizumab and siponimod, in particular, would enter competitive therapeutic classes if approved.