Zevra Therapeutics has announced the commercial availability of MIPLYFFA™ (arimoclomol), marking the first FDA-approved treatment for Niemann-Pick Disease Type C (NPC). This launch represents a significant milestone for patients and families affected by this rare and progressive genetic disorder.
Clinical Significance of MIPLYFFA
MIPLYFFA is indicated for use in combination with miglustat for the treatment of neurological manifestations of NPC in adult and pediatric patients aged 2 years and older. NPC is caused by mutations in the NPC1 or NPC2 genes, leading to abnormal lipid accumulation in cells throughout the body, particularly in the brain. This accumulation results in a range of neurological symptoms, including ataxia, cognitive decline, and seizures.
According to Zevra's chief commercial officer, Josh Schafer, awareness for the new therapy was generated through clinical trials. The company received FDA approval in September for Miplyffa, which addresses the underlying genetic mutations affecting lipid breakdown. While the patient population for NPC is small, the number of diagnosed cases is even smaller, around a few hundred globally. With a first-in-disease approval and recent availability of Miplyffa, Zevra is now ramping up its reach.
Commercialization and Access
MIPLYFFA has been commercially available since November, with a cost exceeding $100,000 per month, translating to over $1 million annually. Despite the high price, early adoption has surpassed expectations, according to Zevra's president and CEO, Neil McFarlane. To support patient access, Zevra has established AmplifyAssist™, a patient support program designed to assist with prescription processes, insurance navigation, and financial assistance.
During a third-quarter earnings call, Zevra announced that it reached 90 patients for Miplyffa and was working through the reimbursement process. According to Schafer, there has been great success in terms of payers’ willingness to pay for Miplyffa. The launch exceeded expectations in terms of interest and demand, and the company's ability to process enrollments and get them authorized for payment from insurance companies and from Medicaid and Medicare.
Ongoing Trials and Expanded Access
Zevra continues to treat patients in its ongoing trials, with 83 patients in its early access program having received MIPLYFFA for up to five years. Of this group, 69 were converted from the early access program to commercially available MIPLYFFA. More than 270 patients have received MIPLYFFA through one of the trials conducted by Zevra, which the company sees as the starting point and accelerator for any rare disease launch.
Addressing Diagnostic Challenges
Zevra is also focusing on increasing awareness and diagnosis of NPC, which can be mistaken for other conditions such as Gaucher disease. As part of its commercialization strategy, Zevra aims to identify misdiagnosed patients to broaden its therapeutic reach. The company is advocating for increased awareness and education among clinicians to improve diagnostic accuracy.
Future Expansion
With the U.S. launch underway, Zevra plans to expand MIPLYFFA to Europe, with regulatory submission expected in 2025. The company also has other assets in its portfolio, including Olpruva for urea cycle disorder and Azstarys for ADHD. Additionally, Zevra has KP1077, targeting rare sleep disorders, in phase 1 and 2 trials, as well as a phase 3 trial for a vascular Ehlers-Danlos syndrome treatment.
