Zevra Therapeutics to Sell FDA Drug Review Voucher for $150M

Zevra Therapeutics, a biotechnology company based in Celebration, Florida, is strategically capitalizing on its recent FDA-approved drug, Miplyffa, designed for Niemann Pick disease type C (NPC), a rare metabolic disorder. With the approval of Miplyffa, Zevra was awarded a priority review voucher, which the company has decided to sell for $150 million. While the buyer of the voucher remains undisclosed, Zevra anticipates that the transaction will be finalized within 30 to 45 days.

Miplyffa's approval marked a significant milestone as it became the first treatment for NPC, a genetic condition that leads to the accumulation of toxic substances in cells. This accumulation results in symptoms such as cognitive impairment and difficulties with speech and swallowing. Shortly after Miplyffa's approval, the FDA also approved another NPC treatment, Aqneursa, developed by IntraBio.

Priority review vouchers, like the one awarded to Zevra, are incentives created to promote the development of drugs targeting rare pediatric diseases. These vouchers can expedite the review process for other rare disease drugs from the usual 10-month period to just six months. Companies often choose to sell these vouchers to major pharmaceutical firms as a means of raising non-dilutive capital.

Zevra is actively exploring the potential benefits of a priority review voucher for its other pipeline assets. Notably, Celiprolol is currently undergoing Phase 3 trials for vascular Ehlers-Danlos syndrome, a rare condition characterized by fragile blood vessels that can lead to severe complications. Additionally, another of Zevra’s drugs, KP1077, has completed Phase 2 testing for idiopathic hypersomnia and Phase 1 testing for narcolepsy. The FDA has indicated that a single pivotal study for KP1077 could suffice for a new drug application for idiopathic hypersomnia, prompting Zevra to evaluate strategic alternatives for advancing this drug's development and commercialization.

In a strategic pivot, Zevra has decided to discontinue its in-house drug discovery efforts and close its laboratories in Iowa and Virginia, opting instead to outsource early-stage research. This shift aligns with the company's focus on late-stage clinical development and commercial opportunities. Alongside Miplyffa, Zevra has also commercialized Olpruva for urea cycle disorders.

The company's strategic transition has led to some organizational changes, including the elimination of specific positions and the departure of key executives, such as co-founder and Chief Development Officer Christal M.M. Mickle and Chief Scientific Officer Sven Guenther, both of whom left the company in December.

In August, Zevra bolstered its financial position by raising $64.5 million through a stock offering to support Miplyffa's commercial launch. By the end of the third quarter of 2024, Zevra reported a cash position of approximately $95 million, estimating that these funds would suffice to support operations until 2027. The sale of the priority review voucher will further enhance the company’s financial standing.

Zevra's Chief Financial Officer, LaDuane Clifton, emphasized that the non-dilutive capital from the voucher sale will strengthen the company's balance sheet by adding $150 million in cash. This will support Zevra’s strategic priorities, which include executing the commercial launches of Miplyffa and Olpruva, and advancing their pipeline of product candidates to address unmet needs within the rare disease community. The company is set to release its fourth quarter and full-year 2024 financial results on March 11.