Zevra has sold its US Food and Drug Administration (FDA) priority review voucher (PRV) for $150m to an undisclosed party.
The US-based company received the PRV when its Niemann-Pick disease type C drug Miplyffa (arimoclomol) won FDA approval in September 2024. Zevra plans to use the funds from the voucher sale to support the commercial launches of Miplyffa and the company’s urea cycle disorder drug Olpruva, which it inherited as part of the $91m acquisition of Acer Therapeutics in 2023.
Niemann-Pick disease type C is a progressive, neurodegenerative lysosomal storage disorder caused by the body’s inability to transport cholesterol and other lipids within cells. This leads to lipid accumulation in tissues, including the brain, resulting in severe physical and cognitive decline.
Miplyffa was the first therapy approved for Niemann-Pick disease type C in the US. It is indicated for use alongside Johnson & Johnson’s (J&J’s) Zavesca (miglustat). Zavesca has long been used as the primary, but off-label treatment for Niemann-Pick disease type C patients in the US. It is however approved for the disease in the UK and Germany. Miplyffa works by inducing the heat shock response to address Niemann-Pick disease type C protein misfolding.
Miplyffa’s approval followed a turbulent regulatory journey. The FDA initially rejected the drug in 2021, issuing a complete response letter (CRL) to its then-owner Orphazyme due to insufficient clinical data. The agency was not convinced by the clinical results and requested additional data. Orphazyme later sold the drug’s rights to KemPharma, which rebranded it as Zevra.
According to GlobalData’s Pharma Intelligence Center, Miplyffa is forecast to generate $273m in global sales by 2030.
GlobalData is the parent company of Pharmaceutical Technology.
The FDA’s PRV programme was introduced to incentivise innovation in areas with limited commercial incentives, such as rare paediatric diseases and neglected tropical diseases.
In February 2024, Valneva sold its PRV for $103m after securing FDA approval for its chikungunya vaccine, Ixchiq, in November 2023. However, not all companies have benefited as expected. In 2023, bluebird bio faced financial and strategic difficulties after failing to secure a PRV for Lyfgenia, a gene therapy it anticipated would qualify for a voucher. The company had pre-arranged a $103m sale of the voucher to Novartis, only for the FDA to deny it, citing similarities to the active ingredient in another of bluebird’s gene therapies called Zynteglo, which had already earned bluebird a PRV on its approval in 2022.
