FDA, Biotech Company Hash Out Criteria for Primary Vitreoretinal Lymphoma Agent’s NDA Resubmission

Biotechnology company Aldeyra Therapeutics, Inc, is developing the investigational drug candidate ADX-2191 as a possible treatment for primary vitreoretinal lymphoma (PVRL), retinitis pigmentosa, and other rare retinal diseases. In a press release, the company announced it has received a Special Protocol Assessment Agreement Letter from the FDA for this agent in PVRL management.

ADX-2191 is a novel intravitreal injection formulation of methotrexate. At this time, no therapies for PVRL are approved by the FDA, but intravitreal injection of compounded methotrexate has been the current standard of care.

“ADX-2191, a novel, vitreous-compatible formulation of methotrexate that is specifically designed for intraocular injection, potentially allows for a reduced injection volume relative to compounding,” explained Aldeyra president and chief executive officer Todd C. Brady, MD, PhD, in the statement announcing the FDA’s grant of the Agreement Letter.

This agent previously received Orphan Drug Designation from the FDA for treatment of PVRL and retinitis pigmentosa. In March 2023, the FDA accepted a literature-based New Drug Application (NDA) for Priority Review, submitted by Aldeyra for therapeutic use of the agent in PVRL. In June of that year, the FDA issued Aldeyra a Complete Response Letter, informing the company that evidence for the agent’s efficacy in the available literature was insufficient.

In the Response Letter, the FDA informed Aldeyra that properly executed, controlled clinical trials would be required for the approval of ADX-2191. The FDA has since reached agreement with the company that the NDA for this agent will be acceptable for resubmission with a single clinical trial backed by literature references.