" class="no-js "lang="en-US"> Aldeyra Therapeutics Submits New Drug Application to the U.S. Food and Drug Administration for the Treatment of Primary Vitreoretinal Lymphoma - Medtech Alert
Friday, May 24, 2024

Aldeyra Therapeutics Submits New Drug Application to the U.S. Food and Drug Administration for the Treatment of Primary Vitreoretinal Lymphoma

Aldeyra Therapeutics has announced the submission of a New Drug Application (NDA) to the US Food and Drug Administration (FDA) for ADX‑2191 (methotrexate injection, USP), an investigational drug candidate, for the treatment of primary vitreoretinal lymphoma, a rare but potentially fatal cancer with no FDA-approved therapy.

The NDA submission is supported by a combination of published literature on the safety and efficacy of methotrexate for the treatment of primary vitreoretinal lymphoma and safety data from the recently completed Phase 3 GUARD Trial of ADX‑2191 for the prevention of 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. In the Phase 3 GUARD Trial, the incidence of punctate keratitis with ADX-2191 administration was observed to be less than that previously reported with intravitreal injection of compounded methotrexate.

“Compounding methotrexate for intravitreal injection, the current standard of care for primary vitreoretinal lymphoma, poses several challenges for physicians and patients, including risk of infection and increased injection volume, potentially leading to ocular hypertension and corneal inflammation,” stated Todd C. Brady, M.D., Ph.D., Aldeyra’s President and Chief Executive Officer. “ADX-2191 is a novel formulation of methotrexate that is designed to be vitreous-compatible and has the potential to be the first marketed drug for patients suffering from primary vitreoretinal lymphoma.”

ADX-2191 has received FDA Orphan Drug Designation for the treatment of primary vitreoretinal lymphoma. In addition, as part of the NDA submission, Aldeyra requested Priority Review Designation, which reduces the review period during which the FDA aims to take action on an NDA to within 6 months, compared to 10 months under standard review.

A Type C meeting with the FDA to discuss the completion of clinical development of ADX-2191 for the prevention of proliferative vitreoretinopathy is planned for the first half of 2023, and results from the Phase 2 clinical trial of ADX-2191 in retinitis pigmentosa are expected in the first half of 2023.

  1. Eloxx Pharmaceuticals Announces Final Data Assessment from Phase 2 Combination Clinical Trial of ELX-02 in Class 1 Cystic Fibrosis Patients Read more
  2. Verge Genomics Announces Positive Safety and Tolerability Data from the Phase 1 Clinical Trial of VRG50635, a Potential Best-in-Class Therapeutic for All Forms of ALS Read more
  3. DEM BioPharma Appoints Wendy Young, Ph.D., to Scientific Advisory Board Read more
  4. Confo Therapeutics Enters into Research Collaboration for GPCR-Targeting Antibody Discovery with AbCellera Read more
  5. Vyriad Announces Expansion of T-Cell Lymphoma Trial at Mayo Clinic Read more