IntraBio Inc., a biopharmaceutical company focused on developing therapies for rare neurological diseases, today announced that the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) has issued a positive opinion recommending approval of AQNEURSA^® (levacetylleucine) for the treatment of Niemann-Pick disease type C (NPC).

"This positive CHMP opinion represents another important milestone in expanding access to AQNEURSA to the global NPC community," said Dr. Marc Patterson, Chief Medical Officer of IntraBio. "The recommendation reflects the strength of our clinical data and the potential for AQNEURSA to be a foundational therapy for NPC, delivering meaningful benefits for patients. We are proud to work alongside the NPC community to bring this long-awaited treatment option to even more families.”

NPC is a rare, inherited lysosomal disorder characterized by progressive neurological deterioration, leading to loss of motor function, difficulties with speech and swallowing, and cognitive decline.³ NPC affects both children and adults, significantly impacting quality of life and daily functioning.^3,4

"For NPC families like mine, this positive opinion brings long-awaited hope for a treatment that can actually offer improvements," said Carmelo Fernández, President of Fundación Niemann-Pick de España. "We have waited years for a therapy that can make a meaningful difference in the lives of people with NPC, and today’s announcement brings us one step closer."

The positive CHMP’s opinion is based on results from IntraBio’s pivotal Phase III randomized, placebo-controlled, clinical trial (IB1001-301; NCT05163288), which evaluated the impact of AQNEURSA on neurological symptoms and functioning in pediatric and adult patients (n=60) with a confirmed diagnosis of NPC. AQNEURSA significantly improved neurological symptoms and functional abilities across its primary and all secondary endpoints within 12 weeks of treatment versus placebo, and was well tolerated throughout the development program.