Avanzanite Bioscience BV, a specialty pharmaceutical company in the development stage, has announced that the European Commission (EC) has granted marketing authorization for AKANTIOR® (polihexanide), a treatment for acanthamoeba keratitis (AK) in adults and children aged 12 and older. This approval follows the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) positive opinion and the Committee for Orphan Medicinal Products (COMP) recommendation. After over 16 years of research and development, AKANTIOR is now the first and only globally authorized treatment for AK.
“Our research indicates that AKANTIOR, when used as a monotherapy with the established dosing schedule, can achieve medical cure rates exceeding 85%. I anticipate this will become the standard of care for this serious disease,” said John Dart, principal investigator of the pivotal Phase III ODAK study and professor at Moorfields Eye Hospital and University College London’s Institute of Ophthalmology. “AKANTIOR has the potential to improve cure rates without surgery, thereby reducing the high rates of morbidity—particularly anxiety, pain, and time away from normal activities—as well as the rates of blindness associated with current unlicensed, often compounded, combination therapies.”
In March 2024, results from a Phase III study involving 135 patients were published in Ophthalmology, demonstrating the efficacy and safety of AKANTIOR. The study revealed that 84.8% of patients treated with AKANTIOR were cured, with 66.7% achieving full vision recovery. Notably, no patients required optical corneal transplantation, and only 7.5% needed therapeutic corneal transplantation.
Juliette Vila Sinclair Spence, a rare disease patient advocate and President of the AK Eye Foundation, expressed optimism about AKANTIOR’s impact. “As an Acanthamoeba Keratitis (AK) Warrior, I believe AKANTIOR will significantly benefit AK patients. When I was diagnosed, there were no licensed treatments like AKANTIOR, and I had to rely on various compounded or off-label eye drops. This approval renews my passion and commitment to raising awareness of this rare eye disease.”
Acanthamoeba keratitis (AK) is a rare, severe parasitic corneal infection caused by the free-living amoeba Acanthamoeba. The disease can lead to blindness and eye loss if not treated urgently and often requires corneal transplants with low graft survival rates. AK primarily affects contact lens wearers, accounting for 50% of blindness in this group. In the absence of anti-amoebic treatment, 80.4% of patients in a historical cohort required surgical intervention.
“This marketing authorization marks a new chapter for AK treatment and a major victory for patients in Europe,” said Adam Plich, Founder and CEO of Avanzanite. “We are thankful to our R&D partners at SIFI, healthcare professionals, and the patients and families who contributed to making this day possible.”
To date, AKANTIOR has been supplied to over 200 patients in 12 countries through a pre-authorization distribution program. “At Avanzanite, we are committed to ensuring broad access to our innovative therapies and look forward to working with local health authorities in 26 European countries to secure sustainable access agreements, enabling us to save the eyes of as many eligible patients as possible,” Plich concluded.
