Zevra Therapeutics, Inc. has announced the U.S. commercial availability of Miplyffa™ (arimoclomol), the first FDA-approved treatment for Niemann-Pick disease type C (NPC). The company reported that the early adoption of Miplyffa has exceeded expectations, and the product is now available for shipment to NPC patients in line with the guidance of eight to 12 weeks post-approval.
Zevra's comprehensive patient support program, Amplifyassist™, is in place to address access barriers for eligible patients and their caregivers. The program offers personalized insurance coverage education and support, copay and alternate funding identification assistance, disease state information, therapy management counseling, and ongoing interactions to facilitate timely prescription refills.
The pivotal phase 3 trial showed that Miplyffa halted disease progression compared to placebo over the one-year duration of the trial when measured by the NPC clinical severity scale. The product was granted breakthrough therapy designation, rare pediatric disease designation, orphan drug designation, and fast track designation by the FDA for the treatment of NPC. Additionally, it was granted orphan medicinal product designation by the European Medicines Agency for the same indication.
Amplifyassist is designed to support the individual needs of eligible patients and their caregivers, offering resources such as personalized insurance coverage education and support, copay and alternate funding identification assistance, disease state information, and therapy management counseling.
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 most common adverse reactions in Miplyffa-treated patients who also received miglustat were upper respiratory tract infection, diarrhea, and decreased weight.
Zevra Therapeutics, Inc. is a commercial-stage rare disease company focusing on bringing life-changing therapeutics to people living with rare diseases through unique, data-driven development and commercialization strategies. The company is committed to assisting those affected by NPC to overcome the barriers and challenges that may impact their treatment journey. Today the company's shares have moved 4.4% to a price of $9.53. Check out the company's full 8-K submission here.
