Press release
Zevra Therapeutics to Present at the 21st Annual WORLDSymposium™
Zevra to receive a 2025 New Treatment Award for MIPLYFFA™ at the 21st Annual WORLDSymposium Eight abstracts discussing data and clinical experience associated
About this update from Zevra Therapeutics, Inc.
[{"type":"text","content":"Zevra to receive a 2025 New Treatment Award for MIPLYFFA™ at the 21st Annual WORLDSymposium Eight abstracts discussing data and clinical experience associated with MIPLYFFA accepted for poster presentation, with one poster selected for oral presentation by Dr. Eugen Mengel CELEBRATION, Fla., Jan. 30, 2025 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra, or the Company), a commercial-stage rare disease therapeutics company, today announced that eight abstracts discussing data and clinical experience associated with MIPLYFFA™ (MY-PLY-FAH) (arimoclomol) have been accepted for poster presentation at the 21st Annual WORLDSymposium™. One poster discussing efficacy results from a 12-month double-blind randomized trial of arimoclomol on the re-scored 4-domain Niemann-Pick disease type C (NPC) Clinical Severity Score (R4DNPCCSS) has been selected for oral presentation by Dr. Eugen Mengel, MD. MIPLYFFA is indicated for use in combination with miglustat for the treatment of neurological manifestations of NPC in adult and pediatric patients 2 years of age and older. In addition, the Company will receive a 2025 New Treatment Award for MIPLYFFA. The New Treatment Award recognizes important achievements in advancing the treatment of lysosomal diseases for products receiving regulatory approval. “The recent FDA approval of MIPLYFFA underscores the persistence required to overcome regulatory challenges and to deliver treatments to people living with rare and ultra-rare diseases,” said Adrian Quartel, MD, FFPM, Zevra’s Chief Medical Officer. “We are honored to receive a 2025 New Treatment Award. WORLDSymposium’s recognition of MIPLYFFA is a testament to the unwavering devotion of physicians, researchers, patient advocacy groups, caregivers, and people living with NPC who have heroically championed MIPLYFFA.” Featured Presentation Details Poster Number:228Title:Efficacy Results from A 12-month Double-blind Randomised Trial of Arimoclomol for Treatment of Niemann-Pick Disease Type C – Presenting A Rescored 4-domain NPC Clinical Severity ScaleDate/Time:Thursday, Feb. 6, 2025, 3:30 p.m. – 5:30 p.m. PT, and oral presentation on Friday, Feb. 7, 2025, 8:30 a.m. PTPresenter: Eugen Mengel, MD, SphinCS GmbH, Institute of Clinical Science for LSD, Hochheim, Germany Poster Presentation Details Poster Number:317Title:Arimoclomol Upregulat...