Advertisment
Zevra Therapeutics presents positive new real-world data on Miplyffa (arimoclomol) in patients with Neimann-Pick Disease Type C at the 22nd Annual WORLDSymposium
Zevra Therapeutics announced the presentation of four posters highlighting positive new data on Miplyffa (arimoclomol) for the treatment of Niemann-Pick Disease Type C (NPC) at the 22nd Annual WORLDSymposium.
“These data highlight MIPLYFFA’s potential to meaningfully stabilize disease progression across a broad spectrum of NPC patients, including adults who have historically had limited clinical data,” said Dr. Adrian Quartel, Zevra’s Chief Medical Officer. “As controlled clinical trial results are reinforced by long-term real-world experience, we continue to strengthen our understanding of MIPLYFFA’s impact and advance our mission to deliver meaningful therapies for people living with rare diseases.”
Key Data Highlights
- 1)“Real-world Safety and Effectiveness of Arimoclomol in Patients with NPC: Outcomes from the U.S. Early Access Program (EAP) Over a 4-Year Period” (Podium Presentation/Poster 41)
- Four years of real-world data from the U.S. EAP demonstrate that arimoclomol was well tolerated and stabilized disease progression in the overall cohort, with changes in clinical severity scores remaining below thresholds for clinically meaningful worsening, supporting sustained benefit across a broad patient population, including adults
- “2) Multi-year Subgroup Analyses of Niemann-Pick Disease Type C Participants Treated with Arimoclomol in the U.S. Early Access Program” (Poster 193)
- Long-term real-world evidence from clinical practice, including data from participants with up to four years of follow-up, demonstrates durable treatment effects of arimoclomol and supports sustained clinical benefit with continued use over time.
- 3) “Efficacy of Arimoclomol Combined with Miglustat at Months 3, 6, 9, and 12 of the Double-blind, Randomized, Placebo-controlled NPC002 Trial” (Poster 250)
- In the post hoc efficacy analysis of the randomized, placebo-controlled NPC002 trial, arimoclomol combined with miglustat demonstrated a statistically significant slowing of disease progression compared to placebo as early as three months after treatment initiation, with sustained and increasing benefit through 12 months, highlighting early onset of clinical effect in patients with NPC.
- 4) “Long-term Safety and Effectiveness of Arimoclomol in Adult and Pediatric Niemann-Pick disease type C Patients in the US Early Access Program (EAP)” (Poster 273)
- Adult NPC patients treated with arimoclomol in the U.S. EAP showed disease stabilization over four years while maintaining a favorable safety profile. This four-year dataset provides the most robust insights to date in this understudied adult NPC population and represents the first published evidence on the impact of arimoclomol in adult NPC patients.
