Aldeyra Therapeutics announced on July 24, 2025, that the European Medicines Agency (EMA) has granted Orphan Designation for ADX-2191. This designation is for the treatment of inherited retinal dystrophies of the rod-dominant phenotype, which includes retinitis pigmentosa. This is a significant regulatory milestone for the investigational drug.
Retinitis pigmentosa is a group of rare genetic eye diseases characterized by retinal cell death and progressive vision loss, affecting over one million people worldwide. There are currently no approved drug treatments for most forms of this serious and incurable condition. The EMA's Orphan Designation highlights the importance of developing new treatment options for these patients.
The Orphan Designation provides Aldeyra with several benefits, including reduced regulatory fees, clinical protocol assistance, research grants, and up to 10 years of market exclusivity in the European Union upon approval. A planned Phase 2/3 clinical trial of ADX-2191 in retinitis pigmentosa is expected to initiate in 2025, building on positive Phase 2 results announced in 2023 that showed improvements in retinal sensitivity.
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