LEXINGTON, Mass. – Aldeyra Therapeutics, Inc. announced that the U.S. Food and Drug Administration (FDA) has granted fast track designation to ADX-2191, an intravitreal methotrexate injection, for the treatment of retinitis pigmentosa, a group of rare genetic eye diseases that lead to retinal cell death and vision loss.
Retinitis pigmentosa affects more than one million people worldwide, yet there are no approved therapies for most forms of the disease.
“Although retinitis pigmentosa is a relentlessly progressive condition that has been recognized for decades as a significant cause of blindness, there are no approved therapies for most forms of the disease,” said Todd C. Brady, M.D., Ph.D., president and CEO of Aldeyra. “Fast Track Designation in conjunction with the previously announced Orphan Drug Designation underscores the potential for ADX-2191 to address an unmet need in the field of ophthalmology and creates the framework for ADX-2191, if approved, to reach patients promptly.”
The designation builds on encouraging Phase 2 results announced in 2023, which showed improvements from baseline in retinal sensitivity following treatment with ADX-2191. Aldeyra plans to launch a Phase 2/3 clinical trial in 2025 to further evaluate the therapy’s potential.
Fast track designation is intended to speed development of therapies for serious or life-threatening conditions that lack effective treatments. It allows for more frequent FDA engagement and may enable rolling review, priority review, or accelerated approval if applicable criteria are met.
