Avanzanite Bioscience Reports European Commission's Authorisation of Orphan Medicinal Product AKANTIOR®

• AKANTIOR (polihexanide) is the first and only medicinal product for the treatment of acanthamoeba keratitis – a rare, severe eye infection that often results in blindness and eye loss
• The marketing authorisation was based on the absolute efficacy established in the phase III ODAK trial and comparison to historical control data. 85% of patients receiving AKANTIOR were cured with only 7% requiring therapeutic cornea transplant and none required optical cornea transplant
• Avanzanite is committed to collaborating with local health authorities to ensure access to AKANTIOR across 26 European countries for eligible patients
• This approval is a quantum leap in Avanzanite’s strategy to expand across Europe, enlarge portfolio, and pu

  rsue ambition as a global rare disease company

Avanzanite Bioscience Reports European Commission's Authorisation of Orphan Medicinal Product AKANTIOR^®

Adam Plich, Founder & CEO
Avanzanite Bioscience B.V.
+31 20 301 21 13
media@avanzanite.com

For medical enquiries, contact medinfo@avanzanite.com

Avanzanite Bioscience B.V., a commercial-stage specialty pharmaceutical company, focused on bringing novel medicines for rare diseases to market, announced today that the European Commission (EC)

=smartlink&url=https%3A%2F%2Fec.europa.eu%2Fhealth%2Fdocuments%2Fcommunity-register%2Fhtml%2Fh1840.htm&esheet=54113227&newsitemid=20240826744342&lan=en-US&anchor=granted+marketing+authorisation&index=1&md5=7af7fbe05ec31a35a50fab96dba8804f" shape="rect">granted marketing authorisation of AKANTIOR^® (polihexanide) for the treatment of acanthamoeba keratitis (AK) in adults and children from 12 years of age. The formal approval by the EC follows previously reported positive opinion of the European Medicines Agency (EMA) Committee for Medicinal Products for Human Use (CHMP) as well as subsequent positive recommendation of the EMA Committee for Orphan Medicinal Products (COMP). Following more than 16 years of research & development (R&D), AKANTIOR is the first and only authorised medicinal product for the treatment of AK globally.

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Adam Plich, Founder & CEO of Avanzanite Bioscience (Photo: Business Wire)

“Our studies have shown that with AKANTIOR, when used as monotherapy with the treatment delivery protocol established for use in the trial, medical cure rates of over 85% can be achieved. As the first authorised drug, I expect this to become the standard of care for this severe disease,” stated John Dart, Principal Investigator of the pivotal phase III ODAK trial, and Professor at the Moorfields Eye Hospital and the University College London Institute of Ophthalmology. “AKANTIOR can be expected to improve the cure rate without surgery, reducing both the high rates of morbidity – principally anxiety, pain, time off normal activities, time to cure – and blindness rates associated with current unlicensed, often compounded, combination therapies.”

In March 2024, results of the 135-patient, phase III, pivotal Orphan Drug for Acanthamoeba Keratitis (ODAK) trial, were published in Ophthalmology, demonstrating AKANTIOR’s absolute efficacy and safety. The trial showed that 84.8% of patients receiving AKANTIOR were cured. Furthermore, 66.7% of patients treated with AKANTIOR achieved full vision restoration with no patients resorting to optical cornea transplant. Only 7.5% of AKANTIOR-treated patients required a therapeutic cornea transplant.

“As an acanthamoeba keratitis (AK) Warrior, I am optimistic that AKANTIOR will have a significant and positive impact on AK patients,” commented Juliette Vila Sinclair Spence, a rare disease patient advocate and the Chairwoman and Founder of AK Eye Foundation – the first global foundation dedicated exclusively to this condition. “When I was diagnosed with AK, for me, there were no authorised drugs or standardised treatment protocols like AKANTIOR available. I had to rely on various compounded or off-label eye drops without a validated protocol. This approval has reignited my passion and commitment to increasing awareness about this rare eye disease.”

Acanthamoeba keratitis (AK) is a rare, severe, and progressive parasitic corneal infection caused by Acanthamoeba, a free-living amoeba. Urgent medical intervention is necessary as the disease leads to blindness and eye loss. AK often requires single or multiple cornea transplant procedures with comparably low graft survival rates. Patients report unbearable pain and extreme light sensitivity and can rarely work or lead normal lives until symptoms resolve, leaving patients with trauma for life. AK primarily affects contact lens wearers and is responsible for 50% of blindness cases in this demographic. In the absence of anti-amoebal treatment, 80.4% of patients require surgery, such as therapeutic cornea transplant, or eye removal in a historical cohort of 56 patients.

“No one fighting such a devastating rare disease should be left behind. This marketing authorisation is the beginning of a new chapter for the treatment of acanthamoeba keratitis (AK) and a monumental victory for AK patients in Europe,” stated Avanzanite’s Founder & CEO, Adam Plich. “We are grateful to our R&D partners at SIFI, the healthcare professionals, as well as the patients and their families who participated in the trials, for the years of dedication and hard work that made this day possible.”

To date, AKANTIOR has been provided to more than 200 patients across 12 countries through a pre-authorisation distribution program. “At Avanzanite, we are committed to supporting broad access to our innovative therapies and look forward to collaborating with local health authorities in 26 European countries to secure sustainable access agreements so we can save the eyes of as many eligible patients as possible,” Plich concluded.

Avanzanite secured exclusive rights to commercialise AKANTIOR in 26 countries across the European Economic Area and Switzerland through a license and supply agreement with SIFI, a leading international ophthalmic company headquartered in Italy. AKANTIOR is the second rare disease medicine commercialised by Avanzanite, expanding both the company’s portfolio and geographical footprint, in support of the ambition to become a global rare disease company.

ABOUT AKANTIOR^®: AKANTIOR (polihexanide) is the first European Commission-approved medicinal product for AK. It is an anti-amoebic polymer granted Orphan Drug Designation (ODD) by the European Medicines Agency ("EMA") and Food and Drug Administration ("FDA"). It acts on both the trophozoites and cysts of the protozoan Acanthamoeba. It is formulated at a unique high 0.8 mg/ml (0.08%) dose strength administered through a validated and standardised, day-only protocol as monotherapy eye drops in single-dose containers. Polihexanide is also being developed for the treatment of fungal keratitis for which it has ODD from both FDA and EMA.

ABOUT AVANZANITE BIOSCIENCE: We believe all patients should benefit from novel orphan or niche medicines regardless of where they live. Our vision is that no one is left behind when facing a debilitating rare disease. We also believe the people who have dedicated their careers to the research & development of novel orphan or niche medicines, deserve to witness how their tremendous efforts impact patients’ lives – the work of these heroes should not lie in vain. We make this happen by acquiring, licensing, or distributing approved or late-stage medicines for rare diseases, unlocking their potential, and commercialising them even where nobody else will. Founded in 2022, Avanzanite is headquartered in Amsterdam, the Netherlands, with commercial infrastructure and technical operations across Europe. Visit www.avanzanite.com for additional information.

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"No one fighting such a devastating rare disease should be left behind."