The latest update is out from Zevra Therapeutics ( (ZVRA) ).
Zevra Therapeutics has announced the FDA’s approval of MIPLYFFA, an oral treatment for Niemann-Pick disease type C (NPC) for patients two years and older, marking a significant breakthrough as the first FDA-approved therapy for this rare and progressive neurodegenerative disease. MIPLYFFA, to be used alongside miglustat, demonstrates Zevra’s commitment to addressing the urgent needs of the NPC community, with the company also launching the AmplifyAssist patient support program. Additionally, the approval comes with a rare pediatric disease priority review voucher, highlighting the drug’s importance in treating this devastating condition.
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