The European Commission (EC) has granted marketing authorisation for IntraBio’s Aqneursa (levacetylleucine) to treat neurological symptoms of Niemann-Pick disease type C (NPC).

The decision follows a positive recommendation from the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA).

Aqneursa is approved across the EU for adults and children aged six years and above who weigh at least 20kg.

The authorisation covers the combined use with miglustat or as a single therapy if miglustat cannot be tolerated.

IntraBio CEO Mallory Factor said: “This approval represents a significant milestone for the NPC community in Europe. We are grateful to the EMA and European Commission for their thorough review and for recognising the clinical value of Aqneursa.

“This decision reflects years of breakthrough scientific work and collaboration with clinicians and patient organisations, and it marks an important step toward expanding access to this therapy for people living with NPC.”

The approval is supported by data from a Phase III randomised, double-blind, placebo-controlled, crossover study in NPC patients.

Results from the study indicated that treatment with Aqneursa provided a statistically significant and clinically important improvement in neurological symptoms and function after 12 weeks.

These improvements were measured by the Scale for the Assessment and Rating of Ataxia and compared with placebo.

In the ongoing open-label extension phase, improvements sustained after the initial 12-week treatment period continued to be observed, reflecting a consistent neuroprotective effect over time.

Observational comparisons with a natural history control group demonstrated that treatment with Aqneursa was linked to an 118% reduction in annual disease progression at one year.

A similar effect was maintained at two years, according to the 5-domain NPC Clinical Severity Scale.

Aqneursa is available as 1g granules for oral suspension. The active ingredient, levacetylleucine, is a modified amino acid targeted at correcting metabolic dysfunction and improving cellular energy in the central nervous system.

The therapy received orphan medicinal product status during its development and is also indicated for similar uses in the US, and has flatulence as its most frequently reported side effect.