U.S. FDA grants orphan drug designation to ADX-2191 from Aldeyra Therapeutics


The U.S. FDA granted orphan drug designation to ADX-2191, methotrexate for intravitreal injection, as treatment for retinitis pigmentosa.

U.S. FDA grants orphan drug designation to ADX-2191 from Aldeyra Therapeutics

On August 4, Aldeyra Therapeutics, Inc. announced that ADX-2191 (methotrexate for intravitreal injection) for the treatment of reinitis pigmentosa (RP) has been granted orphan drug designation by the U.S. Food and Drug Administration (FDA). RP is a clinical collection of rare genetic eye diseases with no approved drug treatments. The group of diseases are characterized by retinal cell death and loss of vision.

“Retinitis pigmentosa is a serious and incurable sight-threatening disease that represents a major unmet need in the field of ophthalmology,” said Todd C. Brady, MD, PhD, President and CEO of Aldeyra. "ADX-2191 has now received orphan designations for three distinct clinical indications, highlighting the broad platform potential of ADX-2191 to treat an array of rare retinal disorders.”

FDA orphan drug designation is a program that financially incentivizes sponsors to develop drugs and biologics for rare diseases and conditions, which are defined as affecting fewer than 200,000 people in the United States. The FDA has previously given ADX-2191 orphan drug designation for the treatment of primary vitreoretinal lymphoma and prevention of proliferative vitreoretinopathy, with additional fast track designation for the latter.

As a biotechnology company, Aldeyra Therapeutics develops novel immune-modulating therapies for ocular and systemic diseases. In addition to the company’s lead product candidates, reproxalap and ADX-629, ADX-2191 (methotrexate for intravitreal injection) is a drug candidate in Phase 3 testing for preventing proliferative vitreoretinopathy. For additional information, visit their website.

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