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Stock impact report

Zevra Therapeutics’ MIPLYFFA™ (arimoclomol) Receives U.S. FDA Approval as Treatment for Niemann-Pick Disease Type C

Zevra Therapeutics, Inc. (ZVRA) 
Company Research Source: GlobeNewswire
MIPLYFFA is the first FDA-approved treatment for Niemann-Pick disease type C (NPC), an ultra-rare and progressive neurodegenerative disease MIPLYFFA is indicated for use in combination with miglustat for the treatment of neurological manifestations of NPC in adults and pediatric patients 2 years of age and older Zevra receives rare pediatric disease priority review voucher in conjunction with approval Company launches AmplifyAssist™ patient support program Conference call and webcast set for 8:00 a.m. EDT on Monday, Sept. 23, 2024 CELEBRATION, Fla., Sept. 20, 2024 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra, or the Company), a commercial-stage rare disease therapeutics company, today announced that the U.S. Food and Drug Administration (FDA) has approved MIPLYFFA™ (MY-PLY-FAH) (arimoclomol) capsules as an orally delivered treatment for Niemann-Pick disease type C (NPC). The first NPC drug approved by the FDA, MIPLYFFA is indicated for use in combination Show less Read more
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