IntraBio, a biopharmaceutical company based in Texas, USA, recently announced that its drug Aqneursa (levoleucovorin) has been approved by the European Commission for the treatment of neurological manifestations of Niemann-Pick disease type C (NPC). Previously, the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) had provided a positive opinion on the drug, laying the groundwork for the final EU approval.

The approved Aqneursa is indicated for adult and pediatric patients aged 6 years and older, weighing at least 20 kilograms. According to the approved regimen, the drug can be used in combination with miglustat or as a monotherapy, particularly for patients who are intolerant to miglustat. This approval represents a new breakthrough in the field of neurological treatment for NPC disease and is expected to significantly improve patients' neurological symptoms and enhance their quality of life.

It is worth noting that the U.S. Food and Drug Administration (FDA) approved the non-nasal gastric formulation of Aqneursa last year. Meanwhile, the FDA also approved Miplyffa (aliglucerase) developed by Zevra Therapeutics for use in combination with the enzyme inhibitor miglustat for the neurological treatment of the same indication. With the approval of Aqneursa in the EU, its application prospects in the global neurological treatment field are set to expand further.