FDA Approves Zevra's Miplyffa for Niemann-Pick Disease Type C — Update

By Colin Kellaher

Zevra Therapeutics has won Food and Drug Administration approval of Miplyffa for Niemann-Pick disease type C, or NPC, making it the first drug cleared in the U.S. for the rare neurodegenerative disorder.

The FDA on Friday said the approval covers Miplyffa in combination with the enzyme inhibitor miglustat to treat neurological symptoms associated with NPC in people ages two and older.

NPC is a rare genetic disease that results in progressive neurological symptoms and organ dysfunction, and the FDA said those affected by the disease only live for about 13 years.

The FDA earlier this year extended its review of Zevra's application seeking approval of Miplyffa, known generically as arimoclomol, after the Celebration, Fla., company submitted additional analyses in response to requests by the agency.

The FDA also convened an advisory committee meeting to discuss the drug, and the panel last month recommended approval of Miplyffa.

Denmark's Orphazyme, which previously owned the arimoclomol program, initially filed for FDA approval of the drug in September 2020, but the agency rejected the application in June 2021.

Zevra acquired all of the assets and operations of Orphazyme related to arimoclomol in May 2022 and resubmitted a new drug application for the drug late last year.

The FDA had previously granted rare-pediatric-disease designation to arimoclomol, and Zevra has said it believes it will be eligible to receive a priority-review voucher that the agency awards upon approval of drugs granted the designation.

The vouchers can be used to obtain priority review for another drug or sold to other companies. France's Ipsen last month said it agreed to sell a voucher to a large global pharmaceutical company for $158 million.

Analysts at William Blair earlier this week noted that it appears unlikely legislation that would renew the voucher program for another five years will proceed to the House floor for a vote before a current sunset provision of Sept. 30, and that a sunsetting of the program could lead to a near-term increase in voucher sale prices.

Trading in shares of Zevra was halted Friday.

Write to Colin Kellaher at colin.kellaher@wsj.com

(END) Dow Jones Newswires

September 20, 2024 12:10 ET (16:10 GMT)

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