Aldeyra Therapeutics announced that the new drug application (NDA) for ADX-2191 (methotrexate injection, USP), a drug candidate for treating primary vitreoretinal lymphoma, has been accepted by the FDA for Priority Review. The Prescription Drug User Fee Act (PDUFA) date has been set for June 21, 2023. The FDA has stated that no potential filing review concerns have been identified.
If approved, ADX-2191 would be the drug available for patients suffering from primary vtreoretinal lymphoma.
“The FDA’s decision to grant Priority Review with a PDUFA date 4 months from NDA acceptance underscores the significant need for an FDA-approved treatment of primary vitreoretinal lymphoma, a rare but potentially fatal cancer,” Todd C. Brady, MD, PhD, Aldeyra’s President and Chief Executive Officer, said in a company news release. “We are working closely with the FDA during the review process to bring ADX‑2191 to patients as quickly as possible, and plan to launch ADX-2191 in the United States in the second half of this year, pending approval by the FDA.”
The submission of the new drug application (NDA) for ADX-2191, which contains methotrexate as an active ingredient, for the treatment of primary vitreoretinal lymphoma, is based on over three decades of published literature on the safety and efficacy of methotrexate, as well as safety data obtained from the phase 3 GUARD trial of ADX-2191 in patients with proliferative vitreoretinopathy.
In the phase 3 GUARD trial, ADX-2191 was well-tolerated, and no safety signals were observed. There were no treatment-emergent serious adverse events. The most commonly observed adverse event associated with ADX-2191 treatment was punctate keratitis, which is a frequent side effect of intravitreal methotrexate, and was mostly mild in severity.
ADX-2191 (methotrexate injection, USP) is a sterile, preservative-free, non-compounded intravitreal formulation of methotrexate, optimized for excipient composition, viscosity, density, tonicity, pH, concentration, and volume of administration.
It is designed to be vitreous-compatible and has potential use for the prevention or treatment of certain rare retinal diseases, including primary vitreoretinal lymphoma, proliferative vitreoretinopathy, and retinitis pigmentosa. The FDA has granted ADX-2191 Orphan Drug Designation for the prevention of proliferative vitreoretinopathy and for the treatment of primary vitreoretinal lymphoma and retinitis pigmentosa.