For Zevra Therapeutics, last year’s approval of the rare lysosomal storage disorder drug Miplyffa appears to be the gift that keeps on giving.
After receiving a rare pediatric disease priority review voucher (PRV) in tandem with Miplyffa’s FDA green light in September, Zevra has locked in a deal to sell the PRV for $150 million.
Zevra did not disclose the identity of the purchaser. The transaction itself is expected to occur within 30 to 45 days, the company said Thursday.
The cash from the sale will be used to fuel R&D, plus the commercial rollouts of Miplyffa and the urea cycle disorder (UCD) medicine Olpruva, which Zevra picked up in its $91 million buyout of Acer Therapeutics in 2023, Zevra’s CFO, LaDuane Clifton, said in a statement.
Miplyffa’s FDA approval last fall made it the first drug specifically cleared in the U.S. to treat Niemann-Pick disease type C (NPC), a rare lysosomal storage disorder that prevents the body from moving and using cholesterol and other lipids in cells. The disease can take a significant toll on patients’ speech, cognition, swallowing, movement and fine motor skills.
Miplyffa is taken orally three times a day in conjunction with the enzyme inhibitor miglustat. Miglustat, which is sold by Johnson & Johnson as Zavesca, has long been used as the primary, albeit off-label, treatment for NPC patients.
Zevra’s first-comer advantage was quickly challenged by Austin, Texas-based IntraBio, which scored an FDA thumbs up for its own NPC drug Aqneursa just one week after Miplyffa’s approval.
Still, Zevra hasn’t been sweating the competition so far.
“It’s really just hard to believe that you have two approvals within one week after literally decades of nothing available—so we’re thrilled for patients and the NPC community,” Zevra’s chief commercial officer, Josh Schafer, said in an interview last year.
Miplyffa’s ability to halt the progression of disease, rather than just treating symptoms, will likely position the drug as the “foundation and the cornerstone” of NPC treatment, Schafer said, adding that he believes additional therapies will be prescribed based on patients’ individual symptoms and needs.
The $150 million price tag for Zevra’s PRV is slightly above the $100 million mark that many vouchers have traded at in the past, though it’s in line with several other recent transactions.
In late November, PTC Therapeutics said it had lined up a $150 million sale of the rare pediatric disease PRV it picked up through the approval of its gene therapy for the ultra-rare disease aromatic L-amino acid decarboxylase (AADC) deficiency.
Earlier that same month, Acadia Pharmaceuticals also bagged a $150 million PRV sale tied to the approval of its Rett syndrome drug Daybue. In Acadia’s case, the company was only able to keep $100 million of the proceeds, with the rest going to the drug's licensor Neuren Pharmaceuticals.
The recent voucher sales by Zevra, PTC and Acadia are likely to be among the last in the childhood rare disease space.
Back in September, the FDA said it would sunset its rare pediatric disease PRV program beginning Dec. 20, 2024.