‘Clinical intuition’ led FDA advisors to back Zevra’s rare disease med

Fierce Biotech August 5, 2024
Annalee Armstrong

Zevra Therapeutics’ rare disease drug seems to be on the path to approval this fall after gaining the backing of an FDA advisory committee, although the experts were split, with some voting yes simply to address an unmet need.

The agency’s Genetic Metabolic Diseases Advisory Committee voted 11 to 5 in favor of arimoclomol on a questions of whether the evidence supports the drug’s efficacy to treat patients with Niemann-Pick disease type C at the Friday meeting. The rare, genetic lysosomal storage disease prevents the body from moving and using cholesterol and other lipids in cells. This leads to a build-up of cholesterol and other lipids in the liver, spleen or lungs.

There are no approved treatments for the disease,...

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