Zevra Therapeutics, Inc. (ZVRA) announced that it will present two posters featuring long-term real-world data on MIPLYFFA (arimoclomol) in patients with Niemann-Pick disease type C (NPC) at the 2026 SSIEM Annual Symposium.
Data from U.S. and German early access programs showed sustained disease stabilization across diverse patient populations, with treatment experience extending up to eight years and a consistent long-term safety profile.
In the U.S. program, disease severity scores remained essentially unchanged through four years of treatment.
Stabilization was observed in both children and adults, while the pivotal trial previously demonstrated a 65% reduction in disease progression with arimoclomol compared with placebo over 12 months.
In the German compassionate use program, 48 patients aged 2 to 63 years received arimoclomol, with no treatment discontinuations due to arimoclomol-related adverse events.
Some patients experienced rapid clinical deterioration after stopping treatment and subsequently restarted therapy.
MIPLYFFA received FDA approval on September 20, 2024, based on data from the pivotal Phase 2/3 NPC-002 trial.
It is approved for use in combination with miglustat to treat neurological manifestations of NPC in adult and pediatric patients aged 2 years and older.
The two posters will be presented on August 26, 2026, during the dedicated poster walk at the SSIEM Annual Symposium.
ZVRA has traded between $7.16 and $15.03 over the past year. The stock closed Monday's trading at $11.65, down 1.85%. In pre-market trading the stock is at $11.80, up 1.29%.
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