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: | 228 |
| Title: | 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 Scale |
| Date/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. PT |
| Presenter: | Eugen Mengel, MD, SphinCS GmbH, Institute of Clinical Science for LSD, Hochheim, Germany |
Poster Presentation Details
| Poster Number: | 317 |
| Title: | Arimoclomol Upregulates Expression of Genes Belonging to the Coordinated Lysosomal Expression and Regulation (CLEAR) Network |
| Date/Time: | Wednesday, Feb. 5, 2025, 3:30 p.m. – 5:30 p.m. PT |
| Presenter: | Hadeel Shammas, PhD, Assistant Director of Research and Scientific Affairs, Zevra Therapeutics, Celebration, FL, USA |
| Poster Number: | 031 |
| Title: | Arimoclomol for the Treatment of Niemann-pick Disease Type C In a Real-world Setting: Long-term Data from an Expanded Access Program in the United States |
| Date/Time: | Thursday, Feb. 6, 2025, 3:30 p.m. – 5:30 p.m. PT |
| Presenter: | Elizabeth Berry-Kravis, MD, PhD, Professor of Pediatrics, Neurological Sciences, and Biochemistry at Rush University Medical Center in Chicago |
| Poster Number: | 135 |
| Title: | Perseverance Is Key for Regulatory Success in Ultra-Rare Diseases – Key Learnings from Arimoclomol’s Regulatory Journey |
| Date/Time: | Thursday, Feb. 6, 2025, 3:30 p.m. – 5:30 p.m. PT |
| Presenter: | Louise Himmelstrup, MSc Pharmacy, Head of Global Regulatory Affairs, Zevra Therapeutics, Celebration, FL, USA |
| Poster Number: | 065 |
| Title: | Safety of Arimoclomol in a Pediatric Sub-Study of Niemann-pick Disease Type C Patients Aged 6 To |