Press release
Zevra Therapeutics’ MIPLYFFA™ (arimoclomol) Receives U.S. FDA Approval as Treatment for Niemann-Pick Disease Type C
MIPLYFFA is the first FDA-approved treatment for Niemann-Pick disease type C (NPC), an ultra-rare and progressive neurodegenerative disease MIPLYFFA is
About this update from Zevra Therapeutics, Inc.
[{"type":"text","content":"MIPLYFFA is the first FDA-approved treatment for Niemann-Pick disease type C (NPC), 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 adults and pediatric patients 2 years of age and older Zevra receives rare pediatric disease priority review voucher in conjunction with approval Company launches AmplifyAssist™ patient support program Conference call and webcast set for 8:00 a.m. EDT on Monday, Sept. 23, 2024 CELEBRATION, Fla., Sept. 20, 2024 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra, or the Company), a commercial-stage rare disease therapeutics company, today announced that the U.S. Food and Drug Administration (FDA) has approved MIPLYFFA™ (MY-PLY-FAH) (arimoclomol) capsules as an orally delivered treatment for Niemann-Pick disease type C (NPC). The first NPC drug approved by the FDA, 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. “NPC is an ultra-rare, relentlessly progressive, degenerative, and fatal disease for which there were no FDA-approved treatment options until today,” said Neil F. McFarlane, President and Chief Executive Officer of Zevra Therapeutics, Inc. “The approval of MIPLYFFA is a monumental milestone for NPC patients and their family members in the U.S. We are immensely grateful for the unwavering support we have received over the years from the families and individuals impacted by NPC as well as the collaborative efforts of advocacy groups, researchers, and clinicians.” In the U.S., it is estimated that 900 people are living with NPC, of which approximately one-third have been diagnosed with this ultra-rare, relentlessly progressive, and fatal neurodegenerative disease.i Both children and adults can be affected by NPC with varying clinical presentations. Characteristically, those living with NPC experience progressive physical and cognitive limitations, with key neurological impairments presenting in speech, cognition, swallowing, ambulation, and fine motor skills. “Until now, those living with ...