Zevra Therapeutics Presents New Data on the Long-Term Safety and Efficacy of Arimoclomol as a Treatment for Niemann-Pick Disease Type C at the SIMD 45th Annual Meeting
CELEBRATION, Fla., April 15, 2024 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra, or the Company), a rare disease therapeutics company, today announced that new long-term real-world data from the arimoclomol expanded access program (EAP: NCT04316637) for the treatment of Niemann-Pick disease type C (NPC) was featured in an oral presentation during the Society for Inherited Metabolic Disorders (SIMD) 45th Annual Meeting taking place at the Sheraton/Le Meridien Charlotte Hotel Complex in Charlotte, NC, from April 14-17, 2024.
- “NPC presents a therapeutic challenge, lacking approved treatment options in the U.S. and invariably leading to progressive loss of independence due to physical and cognitive impairments.
- Among these 26 adults, mean (standard deviation, SD) age at NPC diagnosis and arimoclomol initiation was 23.7 (9.0) and 28.5 (6.5) years, respectively.
- Patients continued arimoclomol treatment for a mean of 21 months (range: 12-32) and 69% (18/26) had recorded miglustat use.
- Dr. Patterson’s talk was entitled “Evaluation of the Long-Term Effect of Arimoclomol in NPC - 48 Months Data from CT-ORZY-NPC-002.”