About Zevra Therapeutics, Inc. - Common Stock (ZVRA)
Zevra Therapeutics, Inc. is a biopharmaceutical company focused on developing innovative therapies for patients with rare diseases and complex disorders. The company leverages advanced scientific research to address unmet medical needs, particularly in the areas of neurological and metabolic conditions. Through its commitment to cutting-edge innovation and patient-centered solutions, Zevra Therapeutics aims to improve the quality of life for individuals affected by challenging health conditions, fostering hope and enhancing treatment options in the biotech landscape. Read More
Zevra Therapeutics (NASDAQGS: ZVRA), a commercial-stage company focused on therapies for rare diseases, announced that four posters on MIPLYFFA (arimoclomol) will be presented at the International Congress of Inborn Errors of Metabolism (ICIEM), held Sept. 2-6, 2025, in Kyoto, Japan. MIPLYFFA, approved in the U.S. for the treatment of Niemann-Pick disease type C (NPC), will be highlighted in a Best Poster award-winning presentation (#BP-19) detailing its unique mechanism of action targeting NPC pathophysiology. Additional presentations will feature positive new data from a pediatric substudy in patients younger than two years old and a prespecified efficacy analysis in patients on miglustat who switched from placebo to MIPLYFFA.
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The FDA has approved Zevra Therapeutics' Miplyffa (arimoclomol) as the first treatment for neurological symptoms of Neimann-Pick disease, type C. This approval follows favorable voting by the Genetic Metabolic Diseases Advisory Committee.
