RARE Daily

With FDA Rejection of Aldeyra’s Experimental Therapy for Lymphoma, Company Explores Expanded Access Program

June 22, 2023

Rare Daily Staff

The U.S. Food & Drug Administration said it would not approve Aldeyra Therapeutic’s experimental therapy ADX-2191 for the treatment of the rare eye cancer primary vitreoretinal lymphoma because of a lack of evidence of effectives due to an inadequate and well-controlled investigations in the literature-based application submission.

Aldeyra said, based on prior discussions with the agency, it did not conduct any clinical trials of ADX-2191 in primary vitreoretinal lymphoma (PVRL).

“While we appreciate the FDA’s position with respect to providing evidence from adequate and controlled trials, we do not currently believe that randomized clinical trials of ADX-2191 in PVRL, a rare and fatal cancer with no approved therapy, are feasible,” said Todd Brady, president and CEO of Aldeyra. “Given the current shortage of methotrexate, the lack of approved therapy for PVRL, and the desire to avoid potential safety risks associated with ocular injection of compounded formulations, we look forward to discussing with the FDA the potential for making ADX-2191 available to PVRL patients under an expanded access program.”

PVRL is a rare, high-grade, aggressive cancer, with a median survival of less than five years.  Methotrexate, the compounded intravitreal injection of which is the standard of care for the treatment of PVRL, is currently in shortage. An expanded access program allows for access to treatment options for serious diseases when other therapeutic options are not available.

ADX-2191 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.

Photo: Todd Brady, president and CEO of Aldeyra

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