Zevra Therapeutics Launches MIPLYFFA™ (arimoclomol) in the U.S. for Niemann-Pick Disease Type C

Zevra Therapeutics, Inc., a company focused on developing treatments for rare diseases, has announced the availability of MIPLYFFA™ (arimoclomol), the first FDA-approved treatment for Niemann-Pick disease type C (NPC). This treatment is now being dispensed through Zevra’s specialty pharmacy. MIPLYFFA is designed to be used alongside miglustat to address the neurological symptoms of NPC in both adults and children aged two and older.

Josh Schafer, Zevra’s Chief Commercial Officer and Executive Vice President of Business Development, shared the company's satisfaction with the early adoption rates of MIPLYFFA. He confirmed that the medication is now ready for shipment to patients, aligning with their projected timeline of eight to twelve weeks post-FDA approval. Schafer emphasized that they are actively receiving prescription enrollments via their patient services program, AmplifyAssist™, and are aiding patients through the benefits verification process to secure the medication.

AmplifyAssist is a comprehensive support program provided by Zevra to assist eligible patients and their caregivers. The program offers various resources such as personalized insurance guidance, copay and funding assistance, disease education, therapy management counseling, and continuous support to ensure timely prescription refills. Patients and caregivers can access more information on MIPLYFFA.com or contact the AmplifyAssist team during business hours.

For patients to begin treatment with MIPLYFFA, they should consult their physician. Physicians can complete an enrollment form on the MIPLYFFA website, and once the prescription is approved, the medication will be delivered to the patient’s home. It is crucial for healthcare providers and patients to refer to the Full Prescribing Information and Instructions for Use to ensure proper administration.

Zevra is dedicated to helping NPC patients overcome obstacles that could affect their treatment journey. The AmplifyAssist program aims to provide tailored support to meet the needs of patients and their caregivers. Resources offered include insurance and copay assistance, disease information, and therapy management counseling. Healthcare providers can initiate the prescription process through the MIPLYFFA website.

MIPLYFFA works by increasing the activation of transcription factors EB (TFEB) and E3 (TFE3), resulting in the upregulation of coordinated lysosomal expression and regulation (CLEAR) genes. It has also been shown to reduce unesterified cholesterol in the lysosomes of human NPC fibroblasts. During a pivotal phase 3 trial, MIPLYFFA demonstrated an ability to halt disease progression compared to a placebo, as measured by the NPC Clinical Severity Scale. The FDA has granted MIPLYFFA several designations, including Breakthrough Therapy, Rare Pediatric Disease, Orphan Drug, and Fast Track. Additionally, the European Medicines Agency (EMA) has recognized MIPLYFFA with Orphan Medicinal Product designation.

Niemann-Pick disease type C (NPC) is a rare, progressive, neurodegenerative disorder that impairs the body's ability to transport cholesterol and lipids, causing their accumulation in various cells, including neurons. The condition is caused by mutations in the NPC1 or NPC2 genes. NPC affects both children and adults, presenting a range of clinical symptoms. The disease often results in physical and cognitive limitations, affecting speech, cognition, swallowing, ambulation, and fine motor skills. The diagnosis can be lengthy, with the disease's progression being irreversible and often leading to early death.

Zevra Therapeutics, Inc. is committed to developing life-changing therapeutics for individuals with rare diseases. The company leverages data and science to create treatment options where none previously existed, aiming to transform the lives of patients with rare conditions.

Zevra also offers expanded access programs in compliance with respective jurisdictional laws and regulations. These programs are subject to the discretion of the treating physician and the company's Expanded Access Program (EAP) policy.