FDA Grants Fast Track Designation to Aldeyra’s ADX-2191 for Retinitis Pigmentosa

The FDA has granted Fast Track Designation for Aldeyra Therapeutics' ADX-2191 (methotrexate intravitreal injection, USP) for the treatment of retinitis pigmentosa (RP).

The FDA’s Fast Track program is designed to expedite the development and review of therapies targeting serious or life-threatening conditions with unmet medical needs. Benefits may include more frequent FDA interactions, eligibility for rolling review of a new drug application (NDA), and the potential for priority review or accelerated approval if criteria are met.

“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, MD, PhD, 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.”

Support for ADX-2191’s potential efficacy in RP comes from results of a phase 2 clinical trial announced in 2023, which demonstrated improvements in retinal sensitivity from baseline following treatment. Aldeyra is preparing to launch a phase 2/3 clinical trial in 2025, which will further evaluate the drug’s safety and efficacy in a broader patient population.