Zevra Therapeutics Receives the US FDA’s Approval of Miplyffa (Arimoclomol Citrate) for Treating Niemann-Pick Disease Type C (NPC)
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The US FDA has awarded priority review voucher & approved Miplyffa (47mg to 124mg, oral, TID) + miglustat to treat neurological manifestations in adults & pediatrics (≥2yrs.) with NPC, with its launch planned within 8-12wks.
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Approval was based on trial of Miplyffa + miglustat vs PBO in NPC patients (2-19yrs.), showing halted disease progression with 0.2 vs 1.9-point reduction at 12mos. Data from a 48mos. OLE trial provided additional evidence of improved outcomes
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Zevra has launched AmplifyAssist, offering insurance education, copay assistance, disease information, therapy management counseling & support for prescription refills for the eligible patients in need
Ref: Zevra Therapeutics | Image: Zevra Therapeutics
Related News:- Zevra Therapeutics Resubmits Arimoclomol’s NDA to the US FDA for Treating Niemann-Pick disease Type C (NPC)
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