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

The U.S. Food and Drug Administration (FDA) has granted Fast Track Designation to Aldeyra Therapeutics’ investigational drug ADX-2191 (methotrexate intravitreal injection, USP) for the treatment of retinitis pigmentosa (RP), a rare, progressive retinal disorder that leads to vision loss and, ultimately, blindness.

Fast Track Designation is reserved for drug candidates targeting serious or life-threatening conditions where there is significant unmet medical need. The designation provides sponsors with benefits such as more frequent interactions with the FDA, eligibility for rolling review of a new drug application (NDA), and the potential for priority review or accelerated approval if clinical criteria are met.

Overview of Retinitis Pigmentosa and ADX-2191’s Development Path

“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,” stated 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.”

Clinical Evidence and Future Development Plans

Support for ADX-2191’s potential efficacy in RP stems from a Phase 2 clinical trial announced in 2023. The trial showed notable improvements in retinal sensitivity from baseline following treatment. Building on these findings, Aldeyra is preparing to initiate a Phase 2/3 clinical trial in 2025, aiming to further evaluate the drug’s safety and efficacy across a broader patient population.