Press release
Zevra Therapeutics Presented New Data for Arimoclomol and OLPRUVA® (Sodium Phenylbutyrate) at the Society for the Study of Inborn Errors of Metabolism (SSIEM) 2024 Annual Symposium
New clinical efficacy and safety data for arimoclomol as a possible treatment for Niemann-Pick disease type C, including from long-term and real-world
About this update from Zevra Therapeutics, Inc.
[{"type":"text","content":"New clinical efficacy and safety data for arimoclomol as a possible treatment for Niemann-Pick disease type C, including from long-term and real-world settings, demonstrate clinically meaningful reduction in disease progression Arimoclomol was well tolerated during the Open Label Extension trial and Early Access Program with no safety signals identified PK modeling studies showed that administration of OLPRUVA while fasting vs fed results in higher drug exposure which may allow for lower effective dosages when taken without food CELEBRATION, Fla., Sept. 06, 2024 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra, or the Company), a rare disease therapeutics company, today announced the presentation of five posters at the Society for the Study of Inborn Errors of Metabolism (SSIEM) 2024 Annual Symposium. Four posters focused on data from multiple studies showing the efficacy and safety of arimoclomol as a treatment for people living with Niemann-Pick disease type C (NPC), and one poster highlighted data from pharmacokinetic modeling studies of OLPRUVA®, a therapy for the long-term management of certain adult and pediatric patients with urea cycle disorders (UCDs) involving deficiencies of carbamylphosphate synthetase (CPS), ornithine transcarbamylase (OTC) or argininosuccinic acid synthetase (AS). “The data collected during the Phase 2/3 study of arimoclomol, including long-term data from Open Label Extension (OLE) and Early Access Program (EAP) participants, add to the large body of evidence that demonstrates arimoclomol’s clinical efficacy and safety as a treatment for people living with NPC,” said Adrian Quartel, M.D., FFPM, Chief Medical Officer of Zevra. “Additionally, we presented PK modeling data from OLPRUVA in both adult and pediatric virtual patients, showing an increase in drug exposure under fasting conditions.” Presentation Details The data for arimoclomol presented at SSIEM is summarized below: Poster Number: 21260 Title:Efficacy Results from a 12-month Double-blind Randomized Trial of Arimoclomol for Treatment of Niemann Pick Disease Type C – presenting an improved 4-Domain NPC Clinical Severity Scale Authors:Marc Patterson, Sven Guenther and Christine i Dali Summary:The treatment effect of arimoclomol was evaluated in a 12-month, double-blind, placebo-controlled clinical trial (NCT02612129) using...