Zevra Therapeutics receives FDA approval for Miplyffa

Zevra Therapeutics announced that the FDA has approved Miplyffa capsules as an orally delivered treatment for Niemann-Pick disease type C. 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 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 CEO of Zevra Therapeutics. “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.”