21 July 2021 - - The US Food and Drug Administration has granted orphan drug designation to US-based Aldeyra Therapeutics, Inc. (NASDAQ: ALDX) for ADX-2191 (methotrexate for intravitreal injection) for the treatment of primary vitreoretinal lymphoma, the company said.

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.

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.

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.