Zevra Therapeutics (ZVRA) announced that MIPLYFFA, the first treatment approved by the U.S. Food and Drug Administration for the treatment of Nieman Pick disease type C, is now commercially available for dispense. 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. “Early adoption of MIPLYFFA has exceeded our expectations, and we are pleased to report that product is now available for shipment to NPC patients. This is in line with the shorter timeframe of our guidance of eight to 12 weeks post-approval,” said Josh Schafer, Chief Commercial Officer and Executive Vice President of Business Development, Zevra Therapeutics. “We continue to receive prescription enrollments through our patient services program, AmplifyAssist, and are supporting patients as they navigate the benefits verification process to obtain MIPLYFFA.”
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