Kubota Vision Receives Orphan Products Clinical Trial Grant for Stargardt Disease Treatment Candidate

Kubota Vision announced that the FDA Office of Orphan Products Development (OOPD) has awarded an orphan products clinical trial grant to support the ongoing phase 3 study of emixustat in Stargardt disease.

Orphan product clinical trial grants support well-controlled studies that will either result in market approval of these products or substantially contribute to the essential data needed for medical product development that will ultimately meet the needs of rare disease patients. The total fund Kubota Vision receives in 3 years will be up to $1.6 million in total.

The study, initiated on November 7, 2018, is a multicenter, randomized, double-masked, and placebo-controlled phase 3 clinical study in which subjects are randomly assigned to emixustat 10 mg or placebo (2:1 ratio) once daily for 24 months. After all subjects have completed the study, the database will be locked and top-line data readout will begin.

“It is a great pleasure and honor to receive this grant from the FDA for our ongoing phase 3 study of emixustat in Stargardt disease,” Ryo Kubota, MD, PhD, Chairman, President and CEO of Kubota Vision Inc., said in a company news release. “The FDA awards this grant to approximately 15% of all applicants each year, whom the FDA recognizes as a great potential to treat rare diseases. We will continue our dedication to advancing this program and enforce our commitment to finding a treatment for this disease.”

The FDA and European Medicines Agency (EMA) granted orphan drug designation to emixustat for the treatment of Stargardt disease.