LEXINGTON, Mass. — Aldeyra Therapeutics, Inc. (Nasdaq: ALDX) announced that the European Medicines Agency (EMA) has granted Orphan Designation for ADX-2191, an intravitreal methotrexate injection, for the treatment of inherited retinal dystrophies of the rod-dominant phenotype, including retinitis pigmentosa.
Retinitis pigmentosa is a group of rare genetic eye diseases marked by progressive retinal cell death and vision loss. Affecting more than one million people globally, the condition currently has no approved drug treatments, making it a significant cause of inherited blindness.
“Retinitis pigmentosa is a serious and incurable sight-threatening disease that represents a major unmet need in the field of ophthalmology,” said Todd C. Brady, M.D., Ph.D., President and CEO of Aldeyra. “The receipt of Orphan Designation from the EMA, in conjunction with the previously announced Orphan Drug Designation from the U.S. Food and Drug Administration, highlights the importance of developing a treatment option for patients suffering from retinitis pigmentosa.”
Support for ADX-2191’s potential efficacy comes from a Phase 2 clinical trial reported in 2023, which showed improvements in retinal sensitivity following treatment. A Phase 2/3 trial is scheduled to begin in 2025 to further evaluate the therapy’s effectiveness in retinitis pigmentosa patients.
The EMA grants orphan designation to therapies intended for rare and life-threatening conditions affecting fewer than five in 10,000 individuals in the European Union. The designation provides several incentives, including reduced regulatory fees, protocol assistance, eligibility for research grants, and up to 10 years of market exclusivity in the EU.
