Zevra Therapeutics to Present New Data on MIPLYFFA and OLPRUVA at Southeastern Regional Genetics Group Annual Meeting

Zevra Therapeutics announced the presentation of three posters on MIPLYFFA and one on OLPRUVA at the Southeastern Regional Genetics Group (SERGG) annual meeting. The posters highlight the efficacy and safety of MIPLYFFA in treating Niemann-Pick disease type C and the ability of OLPRUVA to be administered via gastrostomy tube for urea cycle disorders. The datasets demonstrate sustained disease stabilization in patients treated with MIPLYFFA and the potential for OLPRUVA to improve the management of UCD in some patients.

Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) recently announced the presentation of three posters on MIPLYFFA and one on OLPRUVA at the Southeastern Regional Genetics Group (SERGG) annual meeting. The posters highlighted the efficacy and safety of MIPLYFFA in treating Niemann-Pick disease type C (NPC) and the potential of OLPRUVA for managing urea cycle disorders (UCD) via gastrostomy tube administration.

MIPLYFFA, an approved therapy for NPC, demonstrated sustained disease stabilization in patients over multiple years. The posters presented data from a pivotal trial and an extended 48-month open-label follow-up study, showing a sustained reduction in disease progression for up to five years in over 270 patients worldwide. This aligns with the pivotal Phase 2/3 trial, which showed that MIPLYFFA halted disease progression compared to placebo over a one-year period.

The poster on OLPRUVA highlighted its potential for improving the management of UCD in some patients. The data demonstrated the ability to administer OLPRUVA via gastrostomy tube, offering a new avenue for treatment in patients with UCD.

Zevra's commitment to improving outcomes for individuals with rare diseases was evident in these presentations. The company continues to advance therapies for NPC and UCD, with a focus on long-term efficacy and safety.

References:

[1] https://www.nasdaq.com/articles/zevra-therapeutics-publishes-long-term-efficacy-and-safety-results-miplyffa-niemann-pick