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SIFI Announces the Grant of a Second FDA Orphan Drug Designation for Polihexanide in Fungal Keratitis


This follows the previously-granted ODD for polihexanide in Acanthamoeba Keratitis by the FDA and EMA.
An estimated 15,660 patients with fungal keratitis are diagnosed annually in the US.

SIFI S.p.A. (“SIFI” or the “Company”), a leading international ophthalmic pharmaceutical company, announced today that the U.S. Food and Drug Administration (“FDA”) has approved the company’s Orphan Drug Designation (“ODD”) application for its investigational anti-infective polymer polihexanide for the treatment of fungal keratitis. This is a second orphan ophthalmic indication for the polihexanide Research & Development program.

Fungal keratitis is a severe corneal infection that often results in blindness and eye loss. The disease is most prevalent in tropical and subtropical climates. According to The Lancet ( ), annual global incidence of fungal keratitis is estimated at 1,051,787 cases, with the highest rates in Asia and Latin America. In the U.S., an estimated 15,660 patients annually ¬– most of whom are contact lens wearers or agricultural workers – are diagnosed with fungal keratitis. Only one drug is currently approved for fungal keratitis in the U.S., and around one in three patients fail on the treatment. There are no approved treatments for fungal keratitis in Europe, Japan, Asia and almost anywhere else around the World. In addition to lack of approved medical alternatives, the Company believes the incidence of fungal keratitis may be on the uptick due the global trend in general resistance to anti-fungal therapeutics.

“The opportunity of having a therapeutic option with new mode of action available to treat patients with fungal keratitis is a breakthrough”, says Dr Edward Holland, Professor of Ophthalmology at the University of Cincinnati. “Currently, we have limited topical options and therefore resort to surgery too soon and often with the risk of poor outcomes as medical treatment proves unsuccessful especially for deeper infections”.

“Over the last year, we have accomplished several pivotal milestones towards developing polihexanide first for acanthamoeba keratitis and now for fungal keratitis,” explained Maria-Grazia Mazzone, Executive Director of Business & Portfolio Development at SIFI. “Both are catastrophic cornea infections with dire consequences and, sadly, areas of unmet medical need. For the sake of the patients we are encouraged by the recent decision of the FDA to grant ODD status to this program, which will enable the efficient development and ultimately approval of polihexanide for the treatment of fungal keratitis”, concluded Mazzone.

The FDA’s Orphan Drug Designation program provides orphan status to drugs that are intended for the treatment, prevention, or diagnosis of a rare disease or condition (affecting fewer than 200,000 persons in the U.S). Characteristics of ODD status include seven years of post-approval marketing exclusivity, federal tax credit for expenses incurred in conducting clinical research, and a waiver of Prescription Drug User Fee Act (“PDUFA”) fees.

SIFI is currently evaluating various options for the commercialization of AKANTIOR(R) (polihexanide) globally, including potential out-license agreements outside its core markets.

ABOUT polihexanide: It is an anti-infective polymer which acts via a dual mechanism involving disruption of microorganism cell membranes and selective condensation and/or disruption of microorganism chromosomes by DNA binding. In addition to ODD for fungal keratitis granted by the FDA polihexanide has also ODD for acanthamoeba keratitis granted by the FDA and the European Medicines Agency (“EMA”). For acanthamoeba keratitis, polihexanide is formulated in a 0.8 mg/ml concentration and is being developed under the brand name AKANTIOR(R). Following the announcement in October 2021, that AKANTIOR(R) met primary endpoint in the phase III pivotal trial in acanthamoeba keratitis, SIFI plans to file Marketing Authorisation Application (“MAA”) at the EMA in Q2 2022. SIFI will hold a ‘Type B’ meeting with the FDA for acanthamoeba keratitis in May 2022.

ABOUT SIFI: SIFI is a leading international ophthalmic company, headquartered in Italy, focusing on eye care since 1935. SIFI develops, manufactures and markets innovative therapeutic solutions for patients with ophthalmic conditions. SIFI is fully committed through its R&D to improve the quality of life of patients, exporting treatments to more than 20 countries worldwide with a direct presence in Italy, Spain, France, Romania, Mexico and Turkey.

Brown L., Leck AK, Gichangi M, Burton MJ, Denning DW, The global incidence and diagnosis of fungal keratitis. The Lancet; Oct 2020

Key Contact:
Jelle Kleijn
Global Head of AKANTIOR(R)

Sebastiano Giuffrida
Head of Clinical Development

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Source: SIFI SpA