At A Race Against Blindness, we follow every breakthrough that could bring new possibilities for families living with vision loss. Recently, Aldeyra Therapeutics announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track Designation to ADX-2191, a potential treatment for retinitis pigmentosa (RP).
Why This Matters
Retinitis pigmentosa is a group of rare genetic eye diseases that lead to the death of retinal cells and progressive vision loss. Affecting more than one million people worldwide, RP currently has no approved treatment options for most patients.
About ADX-2191
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What it is: An intravitreal (in-eye) injection of methotrexate, designed specifically for retinal use.
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What makes it unique: Preclinical research suggests ADX-2191 can help clear misfolded rhodopsin, a key factor in certain genetic forms of RP.
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Designations received so far:
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Orphan Drug Designation (U.S. and EU)
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Fast Track Designation (U.S.)
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The Road Ahead
Aldeyra plans to launch a Phase 2/3 clinical trial in 2025 to further evaluate safety and efficacy. Fast Track status means the FDA will work closely with Aldeyra, potentially speeding up development and review. If approved, ADX-2191 could become one of the first therapies available to RP patients across multiple genetic subtypes.
Why We’re Hopeful
For decades, patients with RP have been told there were no treatments available. Each new step—like Fast Track designation—represents progress toward changing that reality. While more research is still needed, this development is another sign of real momentum in the fight against inherited blindness.
At A Race Against Blindness, we’ll continue tracking updates on ADX-2191 and sharing what they could mean for our community.
