Complete Response Letter for ADX 2191, a proposed treatment for primary vitreoretinal lymphoma.- Aldeyra

Although no safety or manufacturing issues with ADX 2191 were identified, the FDA stated that there was a “lack of substantial evidence of effectiveness” due to “a lack of adequate and well-controlled investigations” in the literature-based NDA submission. Based on prior discussions with the FDA, Aldeyra did not conduct any clinical trials of ADX 2191 in 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,” stated Todd C. Brady, M.D., Ph.D., President and Chief Executive Officer of Aldeyra Therapeutics. “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.”.