Source – Aldeyra Therapeutics
On June 21, 2023, Aldeyra disclosed that they have received a Complete Response Letter from the FDA in response to their 505(b)(2) New Drug Application (NDA) for ADX-2191. ADX-2191 is an investigational drug candidate that contains methotrexate for injection, USP, and it is intended for the treatment of primary vitreoretinal lymphoma (PVRL).
It is reasonable to predict that the FDA will raise concerns if a medication application lacks clinical studies. Despite the company’s previous conversations with the government, it was the case for Aldeyra Therapeutics’ application for approval in a rare eye malignancy.
Primary vitreoretinal lymphoma (PVRL) is a rare and aggressive malignancy with a median survival rate of fewer than five years. The business was looking for approval of their therapeutic candidate ADX-2191 in PVRL. Aldeyra decided to schedule a launch for the second half of this year after the FDA in March accepted the company’s application under priority review.
Methotrexate, a cancer treatment initially authorized in the 1950s, is used by ADX-2191 in an injectable form. However, there is now a medication scarcity in the United States, which makes it difficult to conduct clinical studies.
It’s now unclear when the candidate can access the market because the FDA has issued a detailed response letter. However, the business is still optimistic that an Expanded Access Programme would allow ADX-2191 to be administered to patients.
A lack of “adequate and well-controlled investigations” was cited by the FDA as a reason for the “lack of substantial evidence of effectiveness” in Aldeyra’s application for approval, the business stated in a release. According to the company’s statement, no clinical trials were carried out “based on prior discussions” with the FDA.
“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. 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.”
– Todd C. Brady, M.D., Ph.D., President and Chief Executive Officer of Aldeyra Therapeutics
Aldeyra is developing ADX-2191 for the treatment of rare and vision-threatening retinal diseases, including proliferative vitreoretinopathy and retinitis pigmentosa. The company is awaiting the announcement of top-line results from a Phase 2 clinical trial of ADX-2191 in retinitis pigmentosa, scheduled for June 2023. Furthermore, Aldeyra has plans to conduct a Type C meeting with the FDA in the latter half of 2023. The purpose of this meeting is to discuss the progress and completion of the clinical development of ADX-2191 for the prevention of proliferative vitreoretinopathy.
