Zevra: FDA Advisory Committee Votes Favorably on Arimoclomol for NPC Patients

By Stephen Nakrosis

Zevra Therapeutics said a Food and Drug Administration advisory committee voted favorably on Arimoclomol as a treatment for patients with Niemann-Pick Disease Type C.

The FDA Genetic Metabolic Diseases Advisory Committee voted 11-5 that data support Arimoclomol as effective in the treatment of patients with the rare progressive genetic disorder, the company said. While the committee's recommendation will be considered by the FDA, it isn't binding.

Neil McFarlane, president and chief executive officer of Zevra, said the company remains optimistic about the treatment's continued path to approval.

On its website, the National Organization for Rare Disorders said NPC is characterized by an inability of the body to move cholesterol and other fatty substances inside of cells. This can lead to abnormal accumulation of these substances inside the body. Currently, there is no cure for the disease.

Arimoclomol has received orphan-drug designation, fast-track designation, breakthrough-therapy designation and rare pediatric disease designation by the FDA. It also received orphan medicinal product designation for the treatment of NPC by the European Medicines Agency, the company said.

Write to Stephen Nakrosis at stephen.nakrosis@wsj.com

(END) Dow Jones Newswires

August 02, 2024 18:31 ET (22:31 GMT)

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