Aldeyra Therapeutics Receives Orphan Medicinal Product Designation from the European Commission for ADX-2191 Retinal Disease Program
Aldeyra Therapeutics announced that ADX-2191 has been designated an orphan medicinal product by the European Commission for the treatment of retinal detachment. ADX-2191 is an investigational therapy in clinical development in the U.S. for the prevention of proliferative vitreoretinopathy (PVR), a rare but serious sight-threatening retinal disease with no approved treatment. PVR is the leading cause of failure of rhegmatogenous retinal detachment surgery.
“The Commission’s broad orphan medicinal product designation, covering the treatment of retinal detachment, is an important step in advancing the clinical development of ADX-2191 in the European Union,” Todd C. Brady, MD, PhD, President and CEO of Aldeyra, said in a company news release. “ADX-2191 represents a novel therapeutic approach that we believe has the potential to decrease the development of scar tissue that characterizes PVR, breaking the cycle of complex re-attachment surgeries and severe vision loss that often are a consequence of this devastating disease. ADX-2191 has the potential to be the first pharmacological prophylaxis to prevent retinal detachment associated with PVR.”
The Commission’s decision follows a positive opinion on Aldeyra’s application for orphan medicinal product designation from the European Medicines Agency’s Committee for Orphan Medicinal Products. To qualify for the designation, an investigational medicine must be intended to treat a life-threatening or chronically debilitating condition that affects no more than five in 10,000 people in the European Union (EU). Sponsors with medicines that receive this designation are entitled to a range of incentives, including protocol assistance, potential research funding, access to a centralized market authorization procedure, and, if approved, 10 years of EU market exclusivity.
ADX-2191 previously was granted orphan drug status and fast track designation by the FDA for the prevention of PVR. In December 2019, Aldeyra initiated patient enrollment in its phase 3 GUARD Trial, a two-part, multicenter, randomized, controlled, adaptive clinical trial evaluating the efficacy of intravitreal injections of ADX-2191 versus standard-of-care for the prevention of PVR. Aldeyra is exploring additional indications for ADX-2191, including primary intraocular lymphoma, a rare but serious ocular cancer that can affect the retina, uvea, optic nerve, and other ocular structures.
About ADX-2191
ADX-2191, the intravitreal formulation of methotrexate, is designed to inhibit dihydrofolate reductase, an enzyme involved in cellular replication and activation. The observed clinical activity of ADX-2191 in patients with PVR is believed to be the result of down-regulation of aberrant retinal cell proliferation and activity, thereby leading to reduced retinal scarring that is characteristic of PVR. Aldeyra retains an exclusive license to certain patents related to the use of ADX-2191 for the prevention of PVR.