Alkeus Pharmaceuticals, Inc., a biopharmaceutical company dedicated to preserving the sight of individuals impacted by retinal diseases, announced that the Ministry of Health, Labour and Welfare (MHLW) in Japan has granted Orphan Drug Designation for oral gildeuretinol for the treatment of Stargardt disease. Stargardt disease is an inherited retinal disease that often begins in childhood or adolescence, and progressively and irreversibly damages the central vision patients rely on to read, recognize faces, drive and live independently. Gildeuretinol currently is being evaluated in the global Phase 3 NORTHSTAR Study.

“Receiving orphan designation from the Japan Ministry of Health, Labour and Welfare reinforces the significant unmet need for people living with Stargardt disease and the potential of gildeuretinol (ALK-001) to be an important new treatment option," said Michel Dahan, President and CEO of Alkeus Pharmaceuticals. “This designation adds to the growing global momentum behind our program as our pivotal global Phase 3 NORTHSTAR study continues to enroll patients. We remain focused on advancing gildeuretinol with urgency and scientific rigor and are working closely with regulators and the global retina community to advance gildeuretinol as quickly and responsibly as possible.”

In Japan, Orphan Drug Designation is granted to drugs intended for the treatment of rare diseases affecting fewer than 50,000 patients in the country and for which there is a high medical need. Benefits include eligibility for subsidies for development costs and potential market exclusivity for up to 10 years if approved.

Alkeus recently announced dosing of the first patient and is actively enrolling its global Phase 3 NORTHSTAR Study of gildeuretinol as a potential treatment for patients with Stargardt disease. The study is evaluating gildeuretinol’s potential to reduce the growth rate of retinal atrophic lesions and to preserve visual acuity.