Zevra Therapeutics Inc. (ZVRA) said that the U.S. Food and Drug Administration approved MIPLYFFA (MY-PLY-FAH) (arimoclomol) capsules as an orally delivered treatment for Niemann-Pick disease type C.
MIPLYFFA is the first FDA-approved treatment for Niemann-Pick disease type C, 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 adult and pediatric patients 2 years of age and older. In addition, the Company announced that it has received a rare pediatric disease priority review voucher (PRV) in conjunction with the approval.
Zevra noted that it will immediately initiate its launch activities for MIPLYFFA, which is expected to be commercially available in the U.S. in eight to 12 weeks.
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