Zevra Therapeutics has recently resubmitted a new drug application to the U.S. Food and Drug Administration (FDA) for its lead product candidate, arimoclomol, which is intended to treat Niemann-Pick disease type C, a rare neurodegenerative disorder.
The company expects to receive an acknowledgment letter from the FDA, confirming that the resubmission is complete. Within the next 30 days, a target action date will be set by the FDA.
Zevra, based in Celebration, Fla., anticipates that the FDA will classify the resubmission as Class II. This classification means that the agency will review the application within six months of submission.
Initially, Denmark’s Orphazyme owned the arimoclomol program and filed for FDA approval of the drug in September 2020. However, in June 2021, the FDA issued a complete response letter, stating that it would not approve the application in its current form. The agency requested additional qualitative and quantitative evidence.
Zevra acquired all assets and operations of Orphazyme related to arimoclomol in May 2022. The company states that it has addressed the issues raised by the FDA in the complete response letter. Zevra is now accelerating its launch preparations in anticipation of FDA approval.
