FDA Approves Zevra's MIPLYFFA™ for Niemann-Pick Disease Type C

Zevra Therapeutics, Inc., a company specializing in treatments for rare diseases, has announced that the U.S. Food and Drug Administration (FDA) has approved MIPLYFFA™ (arimoclomol) capsules as a treatment for Niemann-Pick disease type C (NPC). This marks the first FDA-approved treatment for NPC, a rare and progressive neurodegenerative disease that affects both children and adults.

MIPLYFFA is designed to be taken in combination with miglustat to manage the neurological symptoms of NPC. The approval is a significant milestone for the estimated 900 people living with NPC in the U.S., about one-third of whom have been diagnosed. NPC leads to progressive physical and cognitive impairments, affecting speech, cognition, swallowing, ambulation, and fine motor skills. The approval of MIPLYFFA offers new hope for those battling this devastating disease.

Neil F. McFarlane, President and CEO of Zevra Therapeutics, expressed his gratitude for the support from families, advocacy groups, researchers, and clinicians. Laurie Turner from the National Niemann-Pick Disease Foundation (NNPDF) and Dr. Elizabeth Berry-Kravis from Rush University Medical Center both highlighted the significance of this FDA approval, emphasizing the urgent need for effective treatments given the complex nature of NPC.

The approval was based on data from a 12-month multicenter, randomized, double-blind, placebo-controlled trial involving NPC patients aged two to 19 years. In this trial, 76% of patients in the MIPLYFFA group and 81% in the placebo group received miglustat as part of their routine care. The trial used the rescored 4-domain NPC Clinical Severity Scale (R4DNPCCSS) to measure effectiveness. Results showed that MIPLYFFA, in combination with miglustat, halted disease progression over 12 months, indicated by a decrease of 0.2 points on the R4DNPCCSS compared to a 1.9-point progression for those treated with miglustat alone. Additional evidence from a 48-month open-label extension study suggested improved outcomes compared to a matched National Institutes of Health NPC natural history cohort.

MIPLYFFA is administered orally three times a day, with dosages ranging from 47 mg to 124 mg based on body weight. Zevra plans to launch MIPLYFFA commercially in the U.S. within the next eight to 12 weeks.

Zevra has also launched AmplifyAssist™, a comprehensive patient support program aimed at helping those affected by NPC manage their treatment journey. The program offers personalized insurance coverage education, copay and alternate funding identification, therapy management counseling, and assistance with prescription refills. Information about the program is available on MIPLYFFA.com.

Zevra Therapeutics will host a conference call and webcast on Monday, September 23, 2024, to discuss the FDA approval of MIPLYFFA. The webcast will be accessible via the Investor Relations section of Zevra’s website.

MIPLYFFA (arimoclomol) works by increasing the activation of transcription factors EB (TFEB) and E3 (TFE3), leading to the upregulation of genes involved in lysosomal function. Clinical trials have shown that the drug reduces unesterified cholesterol in the lysosomes of human NPC fibroblasts. The drug has received multiple designations from the FDA, including Breakthrough Therapy, Rare Pediatric Disease, Orphan Drug, and Fast Track.

Niemann-Pick disease type C (NPC) is a lysosomal storage disorder caused by mutations in the NPC1 or NPC2 genes, leading to an accumulation of cholesterol and other lipids in cells, particularly neurons. This results in progressive physical and cognitive impairments, often leading to early mortality.

Zevra Therapeutics aims to create transformative therapies for rare diseases through data-driven development and commercialization strategies. The company offers expanded access programs for its therapies, subject to local regulations and physician discretion.