Zevra Therapeutics Refiles Arimoclomol Application With FDA

By Colin Kellaher

Zevra Therapeutics has resubmitted a new drug application seeking U.S. Food and Drug Administration approval of its lead product candidate arimoclomol for Niemann-Pick disease type C, a rare neurodegenerative disorder.

Zevra on Wednesday said it expects an acknowledgment letter from the FDA that the resubmission is complete and setting a target action date within 30 days.

The Celebration, Fla., company said it expects the FDA to classify the resubmission as Class II, making it subject to a review period by the agency within six months from the date of submission.

Denmark's Orphazyme, which previously owned the arimoclomol program, initially filed for FDA approval of the drug in September 2020.

However, the FDA in June 2021 issued a so-called complete response letter, indicating that it wouldn't approve the application in its current form and calling for additional qualitative and quantitative evidence.

Zevra, which acquired all of the assets and operations of Orphazyme related to arimoclomol in May 2022, said it believes it has addressed the issues the FDA raised in the complete response letter, and that it is accelerating launch preparations in anticipation of an FDA approval.

Write to Colin Kellaher at [e-mail]

(END) Dow Jones Newswires

December 27, 2023 08:09 ET (13:09 GMT)

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