FDA Grants Priority Review to Aldeyra’s NDA for the Treatment of Primary Vitreoretinal Lymphoma

Rare Daily Staff

The U.S. Food and Drug Administration accepted for Priority Review Aldeyra Therapeutics’ New Drug application  for ADX-2191 for the treatment of primary vitreoretinal lymphoma, a rare and potentially fatal retinal cancer.

The FDA assigned a Prescription Drug User Fee Act action date of June 21, 2023, and noted that no potential filing review issues have been identified.

Primary vitreoretinal lymphoma is an aggressive cancer that is diagnosed in approximately 300 to 600 patients in the United States per year. The median survival for newly diagnosed patients is less than five years. No approved treatments are currently available, though intravitreal injection of compounded methotrexate represents the current standard of care.

ADX-2191 (methotrexate injection, USP) is a sterile, non-compounded intravitreal formulation of methotrexate for the potential prevention or treatment of specific rare retinal diseases, including primary vitreoretinal lymphoma, proliferative vitreoretinopathy, and retinitis pigmentosa. The ADX-2191 intravitreal formulation is preservative-free, is designed to be vitreous-compatible, and is optimized for excipient composition, viscosity, density, tonicity, pH, concentration, and volume of administration. ADX-2191 has received FDA Orphan Drug designation for the prevention of proliferative vitreoretinopathy, and for the treatment of primary vitreoretinal lymphoma and retinitis pigmentosa.

The NDA submission is supported by a combination of more than three decades of published literature on the safety and efficacy of methotrexate, the active ingredient of ADX‑2191, for the treatment of primary vitreoretinal lymphoma, in addition to safety data from the recently completed phase 3 GUARD trial of ADX-2191 in patients with proliferative vitreoretinopathy.

During the phase 3 GUARD Trial, no safety signals were observed, and ADX-2191 was well tolerated; there were no observed treatment-emergent serious adverse events. The most common adverse event associated with ADX‑2191 treatment was punctate keratitis, a frequently observed side effect of intravitreal methotrexate, that was most commonly mild in severity.

“The FDA’s decision to grant Priority Review with a PDUFA date four months from NDA acceptance underscores the significant need for an FDA-approved treatment of primary vitreoretinal lymphoma, a rare but potentially fatal cancer,” said Todd Brady, president and CEO of Aldeyra. “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.”

Photo: Todd Brady, president and CEO of Aldeyra