AQNEURSA® is approved in the European Union for use in adults and children aged 6 years and older weighing at least 20 kg. The approved indication includes use in combination with miglustat, or as monotherapy in patients where miglustat is not tolerated.
“This approval represents a significant milestone for the NPC community in Europe,” said Mallory Factor, Chief Executive Officer of IntraBio. “We are grateful to the EMA and European Commission for their thorough review and for recognizing the clinical value of AQNEURSA®.
This decision reflects years of breakthrough scientific work and collaboration with clinicians and patient organizations, and it marks an important step toward expanding access to this therapy for people living with NPC.”
The approval is based on results from a Phase III randomized, double-blind, placebo-controlled, crossover study in patients with NPC.
In the study, treatment with AQNEURSA® demonstrated a statistically significant and clinically meaningful improvement in neurological signs, symptoms, and functioning after 12 weeks of treatment, as measured by the Scale for the Assessment and Rating of Ataxia (SARA), compared with placebo.1
In the ongoing open-label extension phase of the trial, improvements observed during the initial 12-week study have been sustained,2,3 consistent with a neuroprotective, disease modifying effect over time.
Observational comparisons with a natural history control cohort show that treatment with AQNEURSA® was associated with a 118% reduction in annual disease progression after 1 year, as measured by the 5-domain NPC Clinical Severity Scale (NPC-CSS), and a similar reduction after two-years.2,3
Professor Kyriakos Martakis, Associate Professor in Pediatrics at Justus Liebig University Giessen in Germany, a Principal Investigator for the trial, commented, “Niemann-Pick disease Type C is a rare and relentlessly progressive neurological disorder associated with substantial burden for patients and their families.
The availability of an approved treatment addressing the neurological manifestations of NPC represents an important development for clinicians, but most of all, for individuals affected by this disease across Europe.”
AQNEURSA® is available as 1 g granules for oral suspension. The active substance, levacetylleucine, is a first-in-class modified amino acid delivered to all tissues, including the central nervous system, designed to correct metabolic dysfunction, improve cellular energy production, and target the underlying processes of neurological dysfunction.1,4
Detailed recommendations for the use of AQNEURSA® are described in the Summary of Product Characteristics (SmPC), which is available on the EMA website in all official European Union languages.
About AQNEURSA®
AQNEURSA® (levacetylleucine) is approved in the United States, indicated for the treatment of neurological manifestations of Niemann-Pick disease type C (NPC) in adults and pediatric patients weighing ≥15 kg, and in the European Union for the treatment of neurological manifestations of Niemann-Pick disease Type C, in combination with miglustat or as monotherapy in patients where miglustat is not tolerated, in adults and children aged 6 years and older weighing at least 20 kg.5,6 The most commonly reported adverse reaction with AQNEURSA® was flatulence.
About Niemann-Pick Disease Type C
Niemann-Pick disease Type C (NPC) is a rare (1:100,000 live births), prematurely fatal, autosomal recessive, lysosomal storage disorder.7 The disease presents with systemic, psychiatric, and neurological symptoms, including cerebellar ataxia. NPC is chronic and progressive in nature and is characterized by rapid degeneration of the cerebellum and major organ systems which severely impacts the quality of life.8-10
About IntraBio
IntraBio Inc. is a global biopharmaceutical company that develops and commercializes targeted therapies for rare and common neurological, neurodevelopmental, and mitochondrial diseases. IntraBio's platform technologies result from decades of research and collaboration with universities and institutions worldwide, and leverage the expertise of its scientific founders from the University of Oxford and the University of Munich.
For more information please visit, www.intrabio.com
