Zevra Therapeutics presents new data from arimoclomoal EAP

Zevra Therapeutics announced that new long-term real-world data from the arimoclomol expanded access program for the treatment of Niemann-Pick disease type C was featured in an oral presentation during the Society for Inherited Metabolic Disorders 45th Annual Meeting taking place at the Sheraton/Le Meridien Charlotte Hotel Complex in Charlotte, NC, from April 14-17, 2024. “These real-world data collected in the arimoclomol US EAP represent the first evidence to support the use of arimoclomol in adults with NPC, demonstrating a clinically meaningful slowing of disease progression,” noted Kristina Julich, M.D., Assistant Professor, Department of Neurology, at The University of Texas at Austin, Dell Medical School. “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. We are very pleased with the positive new data that brings hope for a community with a high unmet medical need.” “These observed long-term clinical benefits add to the body of evidence for arimoclomol that supports its tolerability and effectiveness in the treatment of NPC, bringing us one step closer to helping patients suffering from this relentless and fatal disease,” remarked Neil F. McFarlane, President and Chief Executive Officer of Zevra. “We remain committed to continue collaborating closely with the FDA as it reviews arimoclomol’s NDA ahead of our PDUFA date on September 21, 2024.”