Potential oral treatment for geographic atrophy receives fast track designation from the FDA

TAMPA, Fla. — Sirion Therapeutics announced positive 12-month interim results from its phase 2 trial of an oral compound for treating geographic atrophy in patients with age-related macular degeneration, according to a press release from the company.

Based on these results, the U.S. Food and Drug Administration has granted the oral vitamin A-binding protein antagonist, called fenretinide, fast track designation for treating geographic atrophy associated with AMD, the release said.

The interim analysis used retinal photography to compare the growth rate of geographic atrophy lesions in patients treated with once-daily doses of 100 mg of oral fenretinide, 300 mg of oral fenretinide or placebo. Data showed that the 300-mg dose slowed the growth rate of all geographic atrophy lesion sizes; the trend was evident as early as 6 months and increased over time, according to the release.

Among a subpopulation of 78 patients who reached the 18-month study visit, the median lesion growth rate in the 300-mg group was 22.7%, compared with 41.6% in the placebo group, representing a 45% reduction in median lesion growth rate. Subjects in the 100-mg group who had lesions smaller than approximately three disc areas also showed slower lesion growth, suggesting that early intervention may improve outcomes, the release said.

Based on these interim results, Sirion plans to complete the phase 2 study and to work with its advisers and the FDA to design an appropriate phase 3 program for the compound.

Sirion also plans to present data from the interim analysis at the Association for Research in Vision and Ophthalmology meeting in May.