CHMP opinion paves the way for Marketing Authorisation approval by the European Commission, currently anticipated in early August 2024.
When approved, AKANTIOR will be the first licensed medicinal product for the treatment of acanthamoeba keratitis, a rare, severe eye infection that often results in blindness and eye loss.
Phase III ODAK study demonstrated 87% of patients receiving AKANTIOR were cured within a median time of 4 months.
AKANTIOR will be Avanzanite’s second commercialized orphan medicine, expanding the company’s pan-European footprint to 26 countries.

Avanzanite Bioscience B.V. (“Avanzanite” or the “Company”), a commercial-stage specialty pharmaceutical company, solely focused on medicines for rare diseases, announced today that the European Medicines Agency’s (“EMA”) Committee for Medicinal Products for Human Use (“CHMP”) issued a positive opinion recommending Marketing Authorisation of AKANTIOR^® (polihexanide) for the treatment of acanthamoeba keratitis (“AK”) in adults and children from 12 years of age.

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Adam Plich, Founder & CEO, Avanzanite Bioscience B.V. (Photo: Business Wire)

Today’s announcement follows Avanzanite’s licensing and supply agreement with SIFI, a leading Italian ophthalmic healthcare company, that was announced in January 2023, granting Avanzanite the exclusive rights to commercialize AKANTIOR in 26 countries in the European Economic Area and Switzerland.

Avanzanite’s Founder and CEO, Adam Plich, stated, “Avanzanite is a purpose-driven company whose sole mission is to ensure novel medicines reach patients suffering from rare diseases in every market across Europe and beyond. As such, the CHMP positive opinion takes us one step further in our unwavering pursuit of this important goal. No one facing such a monstrous disease should be left behind.” Mr. Plich added, “Through this strategic partnership, Avanzanite is unlocking access to a potential new treatment for those affected by AK in 26 European countries previously unavailable to SIFI.”

This CHMP opinion represents the culmination of more than 15 years of research by SIFI based on a comprehensive data package demonstrating AKANTIOR’s quality, safety, and efficacy. In March 2024, results of the Orphan Drug for Acanthamoeba Keratitis (ODAK) pivotal 135-patient Phase III study were reported and published in Ophthalmology. The trial showed that 86,7% of patients receiving AKANTIOR were cured with a median time to cure of 4,1 months. Furthermore, 66,7% of patients treated with AKANTIOR achieved full vision restoration and only 7,5% required a cornea transplant.

Following the CHMP’s recommendation to approve AKANTIOR for the treatment of AK, the European Commission (EC) will make its final Marketing Authorisation approval decision within approximately 2 months (currently expected in early August 2024). Meanwhile, AKANTIOR remains available through a pre-authorisation distribution program which, to date, has facilitated treatment access to more than 180 patients across 12 European countries.

Following the successful commercialization of Sibnayal^®, AKANTIOR will be the second rare disease medicine commercialized by Avanzanite, expanding the company’s footprint across 26 European countries. In February 2023, Avanzanite announced its exclusive distribution agreement with Paris-based specialty pharmaceutical company Advicenne for Sibnayal, approved in the European Union for the treatment of children and adults with distal renal tubular acidosis (dRTA), a rare genetic or acquired kidney disease. Avanzanite’s dedicated team supplies Sibnayal across 8 countries.

ABOUT AVANZANITE BIOSCIENCE: Founded in 2022, Avanzanite is a privately-funded, commercial-stage, specialty pharmaceutical company solely focused on medicines for rare diseases. At Avanzanite 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 their tremendous efforts impacting 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 and commercializing them even where nobody else will. Headquartered in Amsterdam in the Netherlands, Avanzanite has commercial infrastructure and technical operations across Europe. Please visit www.avanzanite.com for additional information.

ABOUT ACANTHAMOEBA KERATITIS (AK): AK is a rare, severe, and progressive parasitic corneal infection caused by Acanthamoeba, a free-living amoeba. Urgent medical intervention is required as the disease often leads to blindness and eye loss. AK often requires single or multiple corneal 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. While still very rare, the incidence of AK has grown rapidly in recent years. AK primarily affects contact lens wearers, accounting for 50% of blindness among them.

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

View source version on businesswire.com: https://www.businesswire.com/news/home/20240602312116/en/

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

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