Aldeyra Therapeutics (NASDAQ: ALDX) announced that the European Medicines Agency (EMA) has granted Orphan Drug Designation to ADX-2191, its investigational intravitreal methotrexate therapy, for the treatment of primary large B-cell lymphomas of immune-privileged sites, including primary vitreoretinal lymphoma (PVRL). PVRL is a rare, aggressive ocular cancer with no approved therapies in Europe, and treatment options remain limited to systemic chemotherapy or off-label intraocular injections, both of which have drawbacks in safety and efficacy.
This new designation builds on prior regulatory momentum. ADX-2191 already carries orphan status in the United States for both PVRL and retinitis pigmentosa, as well as an EMA designation for inherited retinal dystrophies. Collectively, these designations provide Aldeyra with meaningful incentives, including reduced regulatory fees, support during clinical development, and up to 10 years of market exclusivity in the EU should the drug be approved.
Aldeyra is moving forward with clinical development under a Special Protocol Assessment with the FDA, which outlines the design of a pivotal trial comparing single versus multiple intraocular injections. The study is expected to begin enrolling patients in the second half of 2025, with topline results potentially available by 2026. These efforts are supported by encouraging early data showing ADX-2191’s potential to provide targeted, localized treatment with fewer systemic side effects than existing regimens.
For investors, the EMA orphan designation underscores both the clinical and commercial opportunity for ADX-2191. PVRL affects a very small patient population—estimated at just a few hundred cases annually in Europe—but the lack of effective treatments, coupled with the potential for exclusivity and premium pricing, creates a favorable market dynamic. When combined with Aldeyra’s regulatory progress in the U.S., the designation adds credibility to ADX-2191 as one of the company’s most promising late-stage assets and highlights a clear path toward value-creating milestones over the next 12–18 months.
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