By Stephen NakrosisZevra 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. Neil McFarlane, president and chief executive officer of Zevra, said the company remains optimistic about the treatment's continued path to approval. 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 NewswiresAugust 02, 2024 18:31 ET (22:31 GMT)Copyright (c) 2024 Dow Jones & Company, Inc.

Source: Wall Street Journal August 02, 2024 22:50 UTC