The FDA granted rare pediatric disease and fast track designations to gildeuretinol, an investigational oral therapy for the treatment of Stargardt disease, according to a press release from Alkeus Pharmaceuticals.
“There is enormous unmet need in Stargardt disease because there is currently no approved treatment available to patients,” Seemi Khan, MD, MPH, MBA, chief medical officer of Alkeus Pharmaceuticals, told Healio. “We’re very encouraged about oral gildeuretinol’s potential to be the first therapy in Stargardt. We have generated compelling data in which