Zevra Agrees to Sell PRV for $150 Million

Rare Daily Staff

Zevra Therapeutics said it entered into an agreement to sell its Rare Pediatric Disease Priority Review Voucher for $150 million.

The U.S. Food and Drug Administration granted the PRV to Zevra in September 2024 following the agency’s approval of Miplyffa for use in combination with miglustat for the treatment of neurological manifestations of Niemann-Pick disease type C in adult and pediatric patients 2 years of age and older.

The transaction is subject to customary closing conditions.

The sale follows the recent introduction of bipartisan legislation that seeks to reinstate the voucher program. Without Congressional action, the FDA will not be able to award PRVs to newly designated Rare Pediatric Disease therapies.

The PRV program incentivizes pharmaceutical companies to develop a treatment for rare pediatric diseases. Before a treatment is approved, the company can obtain a Rare Pediatric designation from the FDA. To be eligible, the drug must be eligible for priority review and must be the first approval for the drug’s active ingredient.

After the FDA approves an eligible treatment, the company is issued a PRV that can be used to obtain priority review for a treatment that wouldn’t otherwise qualify. Priority review means the FDA generally reviews a treatment within six months rather than the standard 10-month period. Because the PRVs are transferable, they can provide critical financing to rare disease drug developers who sell them.

“This non-dilutive capital strengthens our balance sheet by adding gross cash proceeds of $150 million, supporting continued investment in our strategic priorities, which include executing the commercial launches of Miplyffa and Olpruva, and advancing our pipeline of product candidates to address unmet needs within the rare disease community,” said LaDuane Clifton, Zevra’s CFO.