FDA approval for Miplyffa (arimcolomol) to treat Nieman Pick disease

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. 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.