-
MIPLYFFA is the first FDA-approved treatment for Niemann-Pick disease type C (NPC), an ultra-rare and progressive neurodegenerative disease
-
MIPLYFFA is indicated for use in combination with miglustat for the treatment of neurological manifestations of NPC in adults and pediatric patients 2 years of age and older
-
Zevra receives rare pediatric disease priority review voucher in conjunction with approval
-
Company launches AmplifyAssist™ patient support program
-
Conference call and webcast set for 8:00 a.m. EDT on Monday, Sept. 23, 2024
CELEBRATION, Fla., Sept. 20, 2024 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra, or the Company), a commercial-stage rare disease therapeutics company, today announced that the U.S. Food and Drug Administration (FDA) has approved MIPLYFFA™ (MY-PLY-FAH) (arimoclomol) capsules as an orally delivered treatment for Niemann-Pick disease type C (NPC). 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.i 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.
“Until now, those living with NPC have had no FDA-approved treatment to combat this devastating disease,” said Laurie Turner, Family Services Manager, National Niemann-Pick Disease Foundation (NNPDF). “For more than 30 years, NNPDF and the community have been working to find treatments for NPC, and we are grateful for the diligence and commitment of the researchers, clinicians, families and Zevra for making this approval possible.”
“The FDA approval of MIPLYFFA marks a significant moment for those living with NPC and the global NPC community,” stated Dr. Elizabeth Berry-Kravis, Professor, Departments of Pediatrics, Neurological Sciences, Anatomy and Cell Biology, Director, RUSH Pediatric Neurosciences F.A.S.T. Center for Translational Research at Rush University Medical Center, “Effective management of NPC requires multiple treatment options due to the complexity of the disease. Until today, there were no approved therapies in the U.S. for NPC. With this labeled indication, patients will now have more access to treatments to tackle this devastating disease.”
The approval of MIPLYFFA for the treatment of NPC is based on the totality of the data in the New Drug Application (NDA), which included additional evidence supporting trial endpoints, FDA-preferred analyses, and additional confirmatory evidence, both clinical and nonclinical. The safety and effectiveness of MIPLYFFA were studied in a 12-month multicenter, randomized, double-blind, placebo-controlled trial in patients with NPC between two and 19 years of age. In this trial, 76% of patients in the MIPLYFFA group and 81% of those in the placebo group received miglustat as part of their routine care. The effectiveness of MIPLYFFA was evaluated using the rescored 4-domain NPC Clinical Severity Scale (R4DNPCCSS). Results from this trial demonstrated:
-
MIPLYFFA, in combination with miglustat, halted disease progression through 12 months of treatment, as demonstrated by a decrease of 0.2 points from baseline on the R4DNPCCSS compared to 1.9 points of progression for patients treated with miglustat alone.
Additional confirmatory evidence included data from a 48-month open-label extension study which suggested improved outcomes when compared to a matched National Institutes of Health NPC natural history cohort.
MIPLYFFA is administered orally, three times a day with or without food, with the exact dosage ranging from 47 mg to 124 mg dependent on body weight, by appropriate patients or caregivers with follow-up from a healthcare provider. Healthcare providers and patients/caregivers should refer to the Full Prescribing Information and Instructions for Use for information on the proper administration of MIPLYFFA.
Zevra will immediately initiate its launch activities for MIPLYFFA, which is expected to be commercially available in the U.S. in eight to 12 weeks.
Launch of AmplifyAssist™ — Comprehensive Support for Patients
Zevra is committed to assisting those whose lives are affected by NPC to overcome the barriers and challenges that may impact their treatment journey. The Company today launched AmplifyAssist, Zevra’s comprehensive patient support program. The mission of the program is to support the individual needs of eligible patients and those who care for them. Available resources include personalized insurance coverage education and support, copay and alternate funding identification assistance for eligible patients, product needs, disease state information and therapy management counseling, and ongoing interactions to address barriers while facilitating timely prescription refills. Information about the program is available at MIPLYFFA.com or via telephone. The AmplifyAssist team can be reached toll-free at (888) 668-4198 from 8 a.m. CT to 6 p.m. CT Monday through Friday. Healthcare providers who want to submit prescriptions can visit MIPLYFFA.com to complete the prescription enrollment form that initiates the process for accessing the treatment.
About MIPLYFFA™ (arimoclomol)
MIPLYFFA (arimoclomol) increases the activation of the transcription factors EB (TFEB) and E3 (TFE3) resulting in the upregulation of coordinated lysosomal expression and regulation (CLEAR) genes. MIPLYFFA has also been shown to reduce unesterified cholesterol in the lysosomes of human NPC fibroblasts. The clinical significance of these findings is not fully understood. MIPLYFFA was granted Breakthrough Therapy designation, Rare Pediatric Disease designation, Orphan Drug designation, and Fast Track designation by the FDA for the treatment of NPC. MIPLYFFA was further granted Orphan Medicinal Product designation by the European Medicines Agency (EMA) for the treatment of NPC.
About Niemann-Pick Disease Type C (NPC)
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. The disease is caused by mutations in the NPC1 or NPC2 genes, which are responsible for making the NPC1 and NPC2 lysosomal proteins. Both children and adults can be affected by NPC with varying clinical presentations. Those living with NPC can lose independence due to physical and cognitive limitations, with key neurological impairments presenting in speech, cognition, swallowing, ambulation, and fine motor skills. Disease diagnosis can often take years, with disease progression being irreversible and often leading to early mortality.
About Zevra Therapeutics, Inc.
Zevra Therapeutics, Inc. is a commercial-stage rare disease company combining science, data, and patient needs to create transformational therapies for diseases with limited or no treatment options. Our mission is to bring life-changing therapeutics to people living with rare diseases. With unique, data-driven development and commercialization strategies, the Company is overcoming complex drug development challenges to make new therapies available to the rare disease community.
Expanded access programs are made available by Zevra Therapeutics, Inc. and its affiliates and are subject to the Company's Expanded Access Program (EAP) policy, as published on its website. Participation in these programs is subject to the laws and regulations of each jurisdiction under which each respective program is operated. Eligibility for participation in any such program is at the treating physician's discretion.
i.Burton et.al., Molecular Genetics and Metabolism Volume 134, Issues 1–2, September–October 2021, Pages 182-187