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FDA grants Aldeyra Therapeutics orphan drug designation for ADX-2191


New intravitreal injection approved to treat retinitis pigmentosa, viteoretinal lymphoma, and other rare cancers

The FDA has granted orphan drug designation to Aldeyra Therapeutics’s methotrexate for intravitreal injection (ADX-2191) for the treatment of patients with retinitis pigmentosa (RP). Currently, no approved drugs are available for the treatment of this rare illness.

Methotrexate is an inhibitor of dihydroflic reductase, which is a protein involved in the cellular activation and replication of tissues.1 RP is characterized by retinal cell death and vision loss. It is estimated to affect 82,000 to 110,000 people in the United States and approximately 1 in 4,000 people worldwide.1

The FDA’s Orphan Drug Program provides financial incentives to companies developing drugs for rare diseases and conditions. Through the program, eligible drugs can be granted tax credits and other benefits.

In related news, the company recently received FDA of ADX-2191 for the treatment of primary viteoretinal lymphoma and other rare cancers.2 It is being studied for the prevention of sclerosing keratomileus, a rare inflammatory disorder that can lead to blindness and severe retinal scarring, in addition to RP.


1. Aldeyra Therapeutics receives orphan drug designation from the U.S. Food and Drug Administration for ADX-2191 to treat retinitis pigmentosa. News release. August 4, 2021. Accessed August 16, 2021. https://ir.aldeyra.com/news-releases/news-release-details/aldeyra-therapeutics-receives-orphan-drug-designation-us-food-0

2. Aldeyra Therapeutics receives orphan drug designation from the U.S. Food and Drug Administration for ADX-2191 to treat primary vitreoretinal lymphoma. News release. July 20, 2021. Accessed August 16, 2021. https://ir.aldeyra.com/news-releases/news-release-details/aldeyra-therapeutics-receives-orphan-drug-designation-us-food

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