Zevra Therapeutics' MIPLYFFA receives FDA approval as first treatment for Niemann-Pick disease type C

ZEVRA THERAPEUTICS, INC.

FDA approved MIPLYFFA (arimoclomol) for NPC in adult and pediatric patients 2 years+; first FDA-approved NPC treatment.
MIPLYFFA is indicated for use in combination with miglustat for neurological manifestations of NPC.
Zevra received a rare pediatric disease priority review voucher (PRV) with the approval.
Launch of AmplifyAssist patient support program; MIPLYFFA expected commercially available in 8-12 weeks.
Conference call and webcast on Sept 23, 2024 at 8 a.m. ET to discuss approval.

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