MIPLYFFA
Latest News
U.S. FDA approves MIPLYFFA™ (arimoclomol) for the Treatment of NPC
Zevra Therapeutics is pleased to share the U.S. Food and Drug Administration (FDA) has approved MIPLYFFA™, for use in combination with miglustat for the treatment of neurological manifestations of Niemann-Pick disease type C (NPC) in adult and pediatric patients 2 years of age and older. MIPLYFFA™ is the first therapy indicated specifically for the treatment of NPC, and is currently the only approved treatment for this disease.
Read the letter to the community.
Read the press release.
Read the FDA press release.
09/20/2024
Update from Zevra Therapeutics
Zevra Therapeutics is pleased to share a letter to the community following Friday’s positive vote, which also includes a link to the press release.
The announcement shares that the U.S. Food and Drug Administration (FDA) Genetic Metabolic Diseases Advisory Committee (GeMDAC) voted favorably that the data support that arimoclomol is effective in the treatment of patients with Niemann-Pick disease type C (NPC).
Zevra will continue to work closely with the FDA and respond to any outstanding questions they may have in the coming weeks. The assigned Prescription Drug User Fee Act (PDUFA) action date for arimoclomol is September 21, 2024.
Read the complete press release.
Read the letter to the community.