Zevra Therapeutics’ MIPLYFFA Earns U.S. FDA Approval for Niemann-Pick Disease Type C Treatment

Zevra Therapeutics, Inc., a company focused on developing therapeutics for rare diseases, has announced that the U.S. Food and Drug Administration (FDA) has approved MIPLYFFA™ (arimoclomol) capsules for the oral treatment of Niemann-Pick disease type C (NPC). This marks the first FDA-approved drug for NPC, indicated for use alongside miglustat in treating neurological manifestations of the disease in patients aged two years and older. The company has also received a rare pediatric disease priority review voucher in connection with this approval.

Neil F. McFarlane, President and CEO of Zevra Therapeutics, stated, “NPC is an ultra-rare and progressive disease with no approved treatments until now. The approval of MIPLYFFA is a major milestone for NPC patients and their families in the U.S. We deeply appreciate the support from families, advocacy groups, researchers, and clinicians throughout this journey.”

In the United States, it is estimated that around 900 individuals are affected by NPC, with about one-third diagnosed with this degenerative neurodegenerative condition. The disease can impact both children and adults, leading to progressive physical and cognitive impairments, including challenges with speech, cognition, swallowing, mobility, and fine motor skills.

Laurie Turner, Family Services Manager for the National Niemann-Pick Disease Foundation, emphasized the significance of this approval, stating, “For over 30 years, the community has worked tirelessly to find treatments for NPC. We are thankful for the commitment of researchers, clinicians, families, and Zevra in bringing this approval to fruition.”

Dr. Elizabeth Berry-Kravis from Rush University Medical Center described the FDA approval as a pivotal moment for the global NPC community, noting that effective management of the disease necessitates multiple treatment options. She highlighted that, until this approval, there were no approved therapies available in the U.S. for NPC, and with this indication, patients will have greater access to potential treatments.

The approval of MIPLYFFA is based on extensive data from the New Drug Application (NDA), which included evidence supporting trial endpoints and analyses. The drug’s safety and efficacy were evaluated in a 12-month multicenter, randomized, double-blind, placebo-controlled trial involving participants aged two to 19. The results showed that MIPLYFFA, when combined with miglustat, halted disease progression, demonstrating a decrease in clinical severity compared to those receiving miglustat alone.

MIPLYFFA is administered orally three times daily, with dosage dependent on body weight. The company plans to launch MIPLYFFA soon, expecting it to be available in the U.S. within eight to twelve weeks.

In addition, Zevra has introduced AmplifyAssist, a comprehensive patient support program designed to assist individuals affected by NPC. This program offers personalized support, including insurance coverage education, copay assistance, product needs, and therapy management counseling, to help navigate the treatment process.

Zevra Therapeutics will host a conference call and audio webcast to discuss the FDA approval of MIPLYFFA, providing information on how to join the call. An archive of the webcast will be available for 90 days following the event.