EU green light to IntraBio’s AQNEURSA for rare neurological disease Niemann-Pick Type C

The approval is grounded in data from a Phase III randomized, double-blind, placebo-controlled, crossover study in NPC patients

Pharma innovator IntraBio has announced that the European Commission has granted marketing authorization for AQNEURSA (levacetylleucine) to treat neurological symptoms of Niemann-Pick Type C (NPC) disease. This follows a positive recommendation from the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP). 

The approval covers adults and children aged 6 years and older weighing at least 20 kg, either in combination with miglustat or as a standalone therapy when 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 grounded in data from a Phase III randomized, double-blind, placebo-controlled, crossover study in NPC patients. AQNEURSA demonstrated statistically significant and clinically meaningful improvements in neurological signs, symptoms, and functioning after 12 weeks, measured by the Scale for the Assessment and Rating of Ataxia (SARA). 

In the ongoing open-label extension, these benefits have been sustained over time, suggesting a neuroprotective, disease-modifying effect. Observational comparisons with a natural history cohort indicate that AQNEURSA reduced annual disease progression by 118% after one year, with similar results after two years, according to the 5-domain NPC Clinical Severity Scale (NPC-CSS). 

Professor Kyriakos Martakis, Associate Professor in Pediatrics at Justus Liebig University Giessen in Germany and a Principal Investigator of the trial, said: “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.”