LEXINGTON, Mass. – Aldeyra Therapeutics, Inc. (Nasdaq: ALDX) (Aldeyra) today announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to the company for ADX-2191 (methotrexate for intravitreal injection) for the treatment of primary vitreoretinal lymphoma (PVRL). There are no approved treatments for PVRL, a rare, aggressive, high-grade cancer that affects approximately 2,800 people in the United States, with approximately 600 new cases diagnosed annually.
“PVRL is an often fatal cancer that creates unique challenges for diagnosis and treatment,” stated Todd C. Brady, M.D., Ph.D., President and CEO of Aldeyra. “The FDA’s orphan drug designation fosters an important clinical development and commercialization pathway for ADX-2191, which represents the first methotrexate preparation specifically formulated for intraocular injection. The designation is another important step forward for our retinal disease program, complementing our ongoing clinical development of ADX-2191 for the prevention of proliferative vitreoretinopathy, the leading cause of failure of retinal reattachment surgery.”
The FDA’s orphan drug designation program is designed to provide financial incentives to sponsors for developing drugs and biologics for rare diseases and conditions, in part defined as affecting fewer than 200,000 people in the United States. Sponsors of designated orphan drugs are eligible for tax credits for clinical trial costs, waiver of the user fee for marketing applications and, upon approval, consideration for seven years of marketing exclusivity.
ADX-2191 (methotrexate for intravitreal injection) inhibits dihydrofolate reductase, an enzyme involved in cellular replication and activation. Methotrexate is the most commonly used intravitreal medication for the treatment of PVRL. ADX-2191 was previously granted orphan drug status and fast track designation by the FDA for the prevention of proliferative vitreoretinopathy, a rare but serious sight-threatening retinal disease with no approved treatment.
About Aldeyra Therapeutics
Aldeyra Therapeutics is a biotechnology company developing novel immune-modulating therapies to treat ocular and systemic diseases. Two of the company’s lead product candidates, reproxalap and ADX-629, target RASP (reactive aldehyde species), which are pre-cytokine, systems-based mediators of inflammation. Reproxalap is being evaluated in Phase 3 clinical trials in patients with dry eye disease and allergic conjunctivitis. The company’s clinical pipeline also includes ADX-2191 (methotrexate for intravitreal injection), a drug candidate in Phase 3 testing for proliferative vitreoretinopathy. For more information, visit https://www.aldeyra.com/ and follow us on LinkedIn, Facebook, and Twitter.
Aldeyra Therapeutics, Inc.
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