Following its new drug application (NDA) submission in December 2022, Aldeyra Therapeutics announced that the FDA has granted the NDA priority review for ADX-2191 (intravitreal methotrexate 0.8%), an investigational drug candidate designed to treat primary vitreoretinal lymphoma (PVL).
Tell me about ADX-2191.
ADX-2191 is a preservative-free intravitreal formulation of methotrexate that is being assessed for the potential prevention or treatment of specific rare retinal diseases such as PVL, proliferative vitreoretinopathy (PVR), and retinitis pigmentosa (RP).
Why priority review?
Aldeyra originally requested priority review—which shortenes the FDA’s typical 10-month review period for market approval to 6 months—due to previously receiving Orphan Drug and Fast track designation for ADX-2191 for the treatment of PVL, PVR, and RP.
What makes it unique?
ADX-2191 is the first sterile, non-compounded formulation of methotrexate designed for intravitreal administration for specific rare retinal diseases.
How did it perform in clinical trials?
The formulation was assessed in the phase 3 GUARD trial in comparison to standard-of-care for patients with PVR over a 6-month period. No safety signals were observed and ADX-2191 was well tolerated. No serious adverse events were reported.
Anything else?
Along with these phase 3 results, the FDA’s priority review acceptance was also based on previously published literature spanning back 30 years regarding methotrexate’s safety and efficacy.
What’s next?
The FDA has set a Prescription Fee Action Date (PFDA) for June 21, 2023. Pending approval, Aldeyra plans on launching ADX-2191 by the second half of 2023.