Zevra Therapeutics’ MIPLYFFA receives FDA approval as first treatment for Niemann-Pick disease

Zevra Therapeutics Inc. has announced U.S. Food and Drug Administration (FDA) approval of MIPLYFFA (arimoclomol), a capsule-based treatment for Niemann-Pick disease type C (NPC), a rare and fatal neurodegenerative disease. This is the first FDA-approved treatment for NPC, which affects around 900 people in the U.S. and has no prior treatment options. MIPLYFFA is designed to be taken orally in combination with miglustat, targeting the neurological manifestations of the disease in both adult and paediatric patients aged two years and older.

NPC is a progressive disorder affecting physical and cognitive functions, such as speech, cognition, and motor skills, often leading to fatal outcomes. Zevra Therapeutics CEO Neil F. McFarlane described the FDA approval as a “monumental milestone” for patients and their families, highlighting the ongoing efforts of advocacy groups and medical professionals in making this treatment possible.

The approval of MIPLYFFA was supported by extensive clinical data, including a multicenter, randomized, double-blind trial in patients aged two to 19 years, which showed significant improvements in halting disease progression. The drug’s effectiveness was evaluated using the rescored 4-domain NPC Clinical Severity Scale (R4DNPCCSS), showing a reduced progression rate compared to patients receiving only miglustat.

Zevra Therapeutics has also received a rare paediatric disease priority review voucher from the FDA as part of this approval. The company plans to launch MIPLYFFA commercially in the U.S. within the next eight to twelve weeks.

MIPLYFFA approval brings new hope to NPC patients

NPC is a highly progressive, life-threatening condition with limited treatment options. The approval of MIPLYFFA signals a breakthrough for the approximately 300 diagnosed U.S. patients, as this new therapy offers a significant intervention against the disease’s neurological damage. Experts such as Dr Elizabeth Berry-Kravis of Rush University Medical Center have emphasised the need for multiple treatment options due to the disease’s complexity. She noted that this approval will provide patients with better access to treatment.

Clinical trials show promise

The effectiveness of MIPLYFFA in treating NPC was demonstrated through a comprehensive 12-month trial. Patients treated with MIPLYFFA and miglustat experienced a 0.2-point decrease in disease severity, compared to a 1.9-point increase in those only receiving miglustat. Further, a 48-month open-label extension study supported these findings by suggesting improved outcomes over time.

This approval also rewards years of dedication by the National Niemann-Pick Disease Foundation (NNPDF) and patient advocacy groups. Laurie Turner, Family Services Manager at NNPDF, expressed gratitude to all those involved in making the approval possible.

What’s next for MIPLYFFA?

Zevra Therapeutics is preparing to launch MIPLYFFA in the U.S., where it will be commercially available within the next few months. The drug will be prescribed in doses ranging from 47 mg to 124 mg based on body weight, to be taken three times a day. Detailed instructions will be available for healthcare providers and caregivers to ensure proper administration.