AInvest Newsletter
Daily stocks & crypto headlines, free to your inbox
Zevra Therapeutics to Present New Data on MIPLYFFA and OLPRUVA at Southeastern Regional Genetics Group Annual Meeting
ByAinvest
Friday, Jul 18, 2025 5:50 pm ET1min read
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
Ainvest News articles are AI-generated using advanced large language model (LLM) technology designed to analyze and synthesize publicly available data and news. While AI tools assist in producing initial drafts and insights, all AI generated content published on Ainvest is subject to comprehensive human editorial review before publication. A designated human editor reviews, verifies, and approves each article for factual accuracy, coherence, and compliance with AInvest Fintech Inc’s editorial and disclosure standards.
Despite these review procedures, the information provided may still contain inaccuracies, incomplete data, or time sensitive references due to the inherent limitations of AI generated analysis and evolving changes. The material is provided strictly for informational and educational purposes and does not constitute personalized investment, legal, or financial advice. Readers should independently verify all facts, figures, and statements before making any decision based on this content.
AInvest Fintech Inc. its affiliates, and partners accept no liability or responsibility for any direct or consequential losses arising from reliance on this content. All articles and analyses are subject to revision, update, or removal without prior notice to maintain accuracy and integrity in our publications.
Comments
No comments yet