Aldeyra Therapeutics Receives Orphan Drug Designation from the FDA for ADX-2191 to Treat Retinitis Pigmentosa

Aldeyra Therapeutics announced that the FDA has granted orphan drug designation for ADX-2191 (methotrexate for intravitreal injection) for the treatment of retinitis pigmentosa (RP).

Methotrexate inhibits dihydrofolic reductase, an enzyme involved in cellular replication and activation. In addition to RP, the FDA has granted orphan drug designation to ADX-2191 for the treatment of primary vitreoretinal lymphoma, a rare, aggressive, high-grade cancer; and both orphan drug and fast track designation to ADX-2191 for the prevention of proliferative vitreoretinopathy, a rare inflammatory disorder of the retina that leads to severe retinal scarring and blindness and is the leading cause of failure of retinal reattachment surgery.

“Retinitis pigmentosa is a serious and incurable sight-threatening disease that represents a major unmet need in the field of ophthalmology,” Todd C. Brady, MD, PhD, President and CEO of Aldeyra, said in a company news release. “ADX-2191 has now received orphan designations for three distinct clinical indications, highlighting the broad platform potential of ADX-2191 to treat an array of rare retinal disorders.”

The FDA’s orphan drug designation program is designed to provide financial incentives to sponsors for developing drugs and biologics for rare diseases and conditions, in part defined as affecting fewer than 200,000 people in the United States. Sponsors of designated orphan drugs are eligible for partial tax credits for clinical trial costs, waiver of the user fee for marketing applications and, upon approval, consideration for seven years of marketing exclusivity.