Zevra Therapeutics is still ramping up sales of its new FDA-approved drug, but the company has already found a way to cash in big on this therapy for a rare metabolic disorder. The priority review voucher awarded alongside the approval of Miplyffa will be sold for $150 million, Zevra announced Thursday.
The buyer of the voucher was not disclosed. Celebration, Florida-based Zevra said it expects the transaction will close within 30 to 45 days.
FDA approval of Miplyffa last September made the Zevra drug the first treatment for Niemann Pick disease type C (NPC), an inherited disorder that leads to defective enzymes important in cellular metabolism. The resulting buildup of toxic substances in cells manifests in symptoms that include impaired cognition and difficulty with speech and swallowing. The week after Miplyffa’s approval, the FDA approved a second NPC treatment, Aqneursa developed by privately held IntraBio.
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Priority review vouchers were created to incentivize R&D of drugs for rare pediatric diseases. Companies awarded a voucher may apply it to another rare disease drug, speeding up its review from the standard 10 months down to six. But these vouchers are transferable and many companies opt to sell them to big pharma companies as a way to raise non-dilutive capital.
Zevra does have assets in its pipeline that could benefit from a priority review voucher. Celiprolol is in Phase 3 testing in vascular Ehlers-Danlos syndrome, a rare disease that causes abnormally fragile blood vessels that can lead to life-threatening complications. Another Zevra drug, KP1077, has completed Phase 2 testing in idiopathic hypersomnia and Phase 1 testing in narcolepsy.
In its report of third quarter 2024 financial results, Zevra said the FDA indicated a single pivotal study for KP1077 would be sufficient to support a new drug application in idiopathic hypersomnia. The company added that it is evaluating strategic alternatives to advance this drug’s development and potential commercialization. Earlier-stage research is no longer part of Zevra’s plans. The company said in the financial report that it is discontinuing in-house drug discovery and closing its labs in Iowa and Virginia. In addition, future early R&D activities will be outsourced.
Going forward, Zevra said it would focus on late-stage clinical development and commercial opportunities. In addition to Miplyffa, the company has commercialized Olpruva for urea cycle disorders. The strategy shift led to the elimination of certain positions in the rank and file as well as the departure of company co-founder and Chief Development Officer Christal M.M. Mickle and Chief Scientific Officer Sven Guenther. Both executives left Zevra in December.
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Last August, Zevra raised $64.5 million in a stock offering to support the planned commercial launch of Miplyffa. As of the end of the third quarter of 2024, Zevra reported its cash position was about $95 million, which it estimated would be sufficient to support operations into 2027. The proceeds from the priority review voucher sale will bring more cash to Zevra’s coffers.
“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,” Zevra Chief Financial Officer LaDuane Clifton said in a prepared statement.
Zevra is scheduled to report fourth quarter and full year 2024 financial results on March 11.
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