The European Commission (EC) has granted marketing authorisation for AKANTIOR (polihexanide), the first and only approved treatment for acanthamoeba keratitis (AK) globally, announced Avanzanite Bioscience B.V., a commercial-stage specialty pharmaceutical company. The approval, which applies to both adults and children aged 12 and over, marks a significant milestone for the treatment of this rare and severe parasitic corneal infection. This decision follows a positive opinion from the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) and a recommendation from the EMA’s Committee for Orphan Medicinal Products (COMP).

Breakthrough in Acanthamoeba Keratitis Treatment

AKANTIOR’s approval comes after more than 16 years of research and development, solidified by the pivotal phase III Orphan Drug for Acanthamoeba Keratitis (ODAK) trial. Published in Ophthalmology in March 2024, the trial results highlighted AKANTIOR’s efficacy, showing that 84.8% of patients were cured, with 66.7% achieving full vision restoration without needing a corneal transplant. Additionally, only 7.5% of those treated with AKANTIOR required therapeutic cornea transplants, a significant reduction compared to historical data where 80.4% of untreated patients required surgical intervention.

Professor John Dart, Principal Investigator of the ODAK trial and a leading expert at Moorfields Eye Hospital and the University College London Institute of Ophthalmology, commented, “Our studies have shown that with AKANTIOR, medical cure rates of over 85% can be achieved when used as monotherapy. As the first authorised drug, I expect this to become the standard of care for this severe disease.”

A Game-Changer for AK Patients

Acanthamoeba keratitis is a rare, progressive infection caused by the Acanthamoeba amoeba, which leads to severe pain, light sensitivity, and often blindness, particularly in contact lens wearers. The disease historically required complex and often unsuccessful treatments, including corneal transplants with low graft survival rates. AKANTIOR’s approval represents a paradigm shift, offering a standardised, effective treatment where none existed before.

Juliette Vila Sinclair Spence, Chairwoman and Founder of AK Eye Foundation, shared her optimism, stating, “When I was diagnosed with AK, there were no authorised drugs like AKANTIOR available. This approval has reignited my passion to increase awareness about this rare eye disease.”

Expanding Access Across Europe

Avanzanite Bioscience, in collaboration with SIFI, an international ophthalmic company, secured exclusive rights to commercialise AKANTIOR in 26 European countries, including those in the European Economic Area and Switzerland. Adam Plich, Founder & CEO of Avanzanite, expressed gratitude to their R&D partners, healthcare professionals, and patients, emphasizing the importance of broad access to this innovative therapy. “We look forward to collaborating with local health authorities to secure sustainable access agreements so we can save the eyes of as many eligible patients as possible,” he stated.

AKANTIOR is not only a breakthrough for AK but also part of Avanzanite’s broader strategy to expand its portfolio and geographical reach, supporting its ambition to become a global leader in rare disease treatments. The drug has been provided to over 200 patients across 12 countries through a pre-authorisation distribution program and continues to represent hope for those affected by this debilitating condition.

  Acanthamoeba Keratitis, AKANTIOR, Avanzanite Bioscience, EMA, European Commission, Orphan Drug, Rare diseases