EMA’s Committee for Medicinal Products for Human Use (CHMP) has adopted a negative opinion on the MAA related to Zevra Therapeutics Inc.’s arimoclomol for Niemann-Pick disease type C (NPC). Boston-based Zevra said it will ask for a re-examination after CHMP gatekeepers ruled that efficacy has not been sufficiently proven. The U.S. FDA green-lighted the heat shock protein amplifier (used in combination with miglustat) as Miplyffa in September 2024. Through its global expanded access program, Zevra is helping eligible NPC patients get access to arimoclomol and thereby generate additional real-world evidence of its clinical impact. Ltd. closed an $8 million series A financing round to support moving lead candidate TAVST-01 into the clinic.