The FDA has approved Zevra Therapeutics' Miplyffa (arimoclomol) as the first treatment for neurological symptoms of Neimann-Pick disease, type C. This approval follows favorable voting by the Genetic Metabolic Diseases Advisory Committee.
FDA panel supports Zevra Therapeutics' arimoclomol for Niemann-Pick disease type C with an 11-5 vote. William Blair boosts success probability to 90%, highlighting Zevra's growth and balanced rare disease portfolio. Key decisions and market opportunities ahead in 2024.
FDA's Genetic Metabolic Diseases Advisory Committee will review Zevra Therapeutics' arimoclomol for Niemann-Pick disease type C on August 2. PDUFA decision expected by September 21.
Companies Reporting Before The Bell • Universal Stainless (NASDAQ:USAP) is expected to report quarterly earnings at $0.32 per share on revenue of $78.65 million.
Top Wall Street analysts changed their outlook on these top names. For a complete view of all analyst rating changes, including upgrades and downgrades, please see our analyst ratings page.
