Zevra Therapeutics, Inc. (NASDAQGS: ZVRA) has announced the U.S. Food and Drug Administration (FDA) approval of Miplyffa™ (arimoclomol) capsules as an orally delivered treatment for Niemann-Pick Disease Type C (NPC). This approval marks a significant milestone as Miplyffa is the first FDA-approved treatment for this ultra-rare and progressive neurodegenerative disease.
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. The approval was based on the totality of the data in the new drug application, including evidence from a 12-month multicenter, randomized, double-blind, placebo-controlled trial in patients with NPC between two and 19 years of age.
The trial results demonstrated that Miplyffa, in combination with miglustat, halted disease progression through 12 months of treatment, with a decrease of 0.2 points from baseline on the Rescored 4-Domain NPC Clinical Severity Scale (R4DNPCCSS) compared to 1.9 points of progression for patients treated with miglustat alone. Additionally, data from a 48-month open-label extension study suggested improved outcomes when compared to a matched national institutes of health NPC natural history cohort.
Furthermore, Miplyffa has been granted breakthrough therapy designation, rare pediatric disease designation, orphan drug designation, and fast track designation by the FDA for the treatment of NPC. It has also received orphan medicinal product designation by the European Medicines Agency (EMA) for the treatment of NPC.
In addition to the approval, Zevra Therapeutics has launched the AmplifyAssist™ patient support program, which aims to provide comprehensive support for eligible patients and their caregivers. The program offers resources including personalized insurance coverage education and support, copay and alternate funding identification assistance, disease state information and therapy management counseling, and ongoing interactions to address barriers while facilitating timely prescription refills.
Miplyffa is expected to be commercially available in the U.S. within eight to 12 weeks, and healthcare providers can visit miplyffa.com to complete the prescription enrollment form that initiates the process for accessing the treatment.
Zevra Therapeutics, Inc. will host a conference call and audio webcast at 8 a.m. ET on Monday, September 23, 2024, to discuss the FDA approval of Miplyffa. An archive of the webcast will be available for ninety (90) days beginning at approximately 9 a.m. ET on September 23, 2024.
Niemann-Pick Disease Type C (NPC) is an ultra-rare, progressive, and neurodegenerative lysosomal storage disorder characterized by an inability of the body to transport cholesterol and other lipids within the cell, leading to an accumulation of these substances in various cell types, including neurons. It is caused by mutations in the NPC1 or NPC2 genes, leading to varying clinical presentations in both children and adults.
Zevra Therapeutics, Inc. is a commercial-stage rare disease company focused on bringing life-changing therapeutics to people living with rare diseases. With the FDA approval of Miplyffa, the company has achieved a significant milestone in providing a treatment option for NPC, addressing a critical unmet medical need for patients and their families. The market has reacted to these announcements by moving the company's shares 0.5% to a price of $8.06. For more information, read the company's full 8-K submission here.
