Friday, the FDA’s Genetic Metabolic Diseases Advisory Committee (GeMDAC) voted in favor of Zevra Therapeutics, Inc.’s (NASDAQ:ZVRA) arimoclomol.

The committee voted favorably (11 yes, 5 no) that the data supports that arimoclomol is effective in treating patients with Niemann-Pick disease type C (NPC), a rare progressive genetic disorder characterized by an inability of the body to transport cholesterol and other fatty substances (lipids) inside of cells.

The committee’s recommendation will be considered by the FDA as it completes its independent review of the arimoclomol NDA; however, the feedback from the GeMDAC is not binding upon the agency.

The arimoclomol NDA has been assigned a September 21 Prescription Drug User Fee Act (PDUFA) action date.

In 2021, the FDA issued a complete response letter to arimoclomol as a treatment option for Niemann-Pick disease type C.

The CRL asked for additional qualitative and quantitative evidence to further support the validity and interpretation of the 5-domain NPC Clinical Severity Scale, particularly the swallow domain.

Zevra Therapeutics resubmitted its New Drug Application (NDA) for arimoclomol to the FDA in December 2023.

William Blair has increased the probability of success of arimoclomol in NPC to 90% from 80%, citing the positive AdCom vote.

The analyst highlights that Zevra is evolving into a fully integrated biotechnology company with a balanced portfolio of marketed products and late-stage development assets focused on rare diseases.

“We believe the launch of Olpruva in 2024 represents an attractive market opportunity where the company can see some early commercial success ahead of the potential approval of its second asset,arimoclomol, which is currently under FDA review for NPC,” the analyst adds.

In December 2022, the FDA approved Olpruva for urea cycle disorders.

2024 represents a significant year for Zevra as new management continues to guide the company through a transformational period with one launch underway, a potential approval and second launch later in the year, and updates from the clinical pipeline, William Blair notes and keeps the Outperform rating.

Price Action: ZVRA stock is up 16.7% at $7.35 during the premarket session at last check Monday.

Photo via Shutterstock

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