Zevra Therapeutics presents MIPLYFFA data at NNPDF Conference, FDA-approved NPC treatment.

Zevra Therapeutics announced oral and poster presentations on MIPLYFFA (arimoclomol) at the National Niemann Pick Disease Foundation Conference. MIPLYFFA, the first FDA-approved treatment for Niemann-Pick disease type C, is indicated for use in combination with miglustat for adult and pediatric patients 2 years of age and older.

Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) has announced that its FDA-approved treatment, MIPLYFFA (arimoclomol), will be featured in three presentations at the National Niemann Pick Disease Foundation Conference, scheduled for July 10-13, 2025, in Concord, North Carolina. The conference presentations will highlight clinical data demonstrating MIPLYFFA's efficacy and safety profile when used in combination with miglustat to treat Niemann-Pick disease type C (NPC) [1].

MIPLYFFA is the first FDA-approved treatment for NPC, indicated for use with miglustat in patients 2 years and older. The presentations will include data from pivotal trials showing that MIPLYFFA, combined with miglustat, halted disease progression at 12 months. Long-term data from a 48-month open-label extension study further validated the treatment's effectiveness and safety profile [1].

The drug's mechanism of action involves upregulation of CLEAR genes and improvement of lysosomal function, targeting the underlying pathophysiology of NPC. The presentations will also showcase in vitro data confirming multiple mechanistic pathways, including increased NPC1 protein processing and enhanced cholesterol clearance from lysosomes [1].

Zevra's presentations on MIPLYFFA represent a significant milestone in rare disease treatment. As the first FDA-approved therapy for NPC, MIPLYFFA addresses a critical unmet need in a serious lysosomal storage disorder with limited treatment options. The pivotal study data being presented demonstrates that MIPLYFFA, when used with miglustat, halted disease progression at 12 months compared to placebo—a remarkable outcome for a progressive neurological disease [1].

The drug's safety profile includes hypersensitivity reactions and potential embryofetal toxicity, which are manageable concerns for a treatment addressing a serious progressive disease. Zevra now has a commercial-stage product with orphan designation both in the US and Europe, creating a significant market opportunity despite the small patient population [1].

References:
[1] https://www.stocktitan.net/news/ZVRA/zevra-therapeutics-announces-miplyffa-arimoclomol-featured-in-ydi128xzk767.html