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Acanthamoeba keratitis (AK) is a rare parasitic infectious disease of the eye, most frequently affecting individuals who wear disposable contact lenses. If not treated promptly, it can lead to visual impairment and even blindness. As a result, it is of primary importance to develop and establish an efficacious treatment for AK.
Retrospective studies had shown positive clinical outcomes of AK patients treated with the unlicensed drug polihexanide (PHMB), an antiseptic that is able to penetrate AK’s tough spores.
With this in mind, the partners of the EU-funded project ODAK decided to implement a clinical trial to demonstrate the efficacy of PHMB, designated as an orphan drug for AK in 2017.
Apart from testing the quality, safety and efficacy of PHMB, different formulations were tested in terms of efficacy and ocular tolerance in preclinical models. ODAK partners were able to optimise the formulation, which showed a high therapeutic effect in a pivotal phase III clinical trial.
Regulatory approval
Phase III trials launched at the end of the ODAK project allowed the drug to enter human trials. The first 23 patients were recruited in Italy, Poland and the United Kingdom during the course of the project. “It’s a rare disease, so it’s hard to find patients, which is why the trial took a long time,” explains Ritchie Head, director of ODAK partner Ceratium.
Since the project concluded in 2017, results from the ODAK project have helped project partner and leading international ophthalmic company SIFI take the PHMB formulation, branded as AKANTIOR®, closer to becoming the first approved orphan medicinal product for AK.
In June 2022, SIFI announced that the European Medicines Agency had validated its marketing authorisation application for AKANTIOR®. John Dart, principal investigator of the phase III clinical trial and clinical professor at University College London in the United Kingdom, notes: “AK is one of the most severe and painful eye infections and also one for which there is no licensed therapy. Most patients require months of treatment and 25 % lose useful vision.”
He adds: “Our findings show that AKANTIOR® will provide a majority of AK patients the prospect of recovering their vision and quality of life; I hope that this will soon become the standard of care for treatment of this disease.”
It is expected that AKANTIOR® will be launched commercially across Europe in 2023, with patients able to seek treatment even sooner through a pan-European early access programme.