Aldeyra Therapeutics (NASDAQ:ALDX) announces that the U.S. FDA has granted orphan drug designation for ADX-2191 (methotrexate for intravitreal injection) for the treatment of retinitis pigmentosa (RP), a group of rare genetic eye diseases.
Shares up more than 6% premarket.
There are no approved drug treatments for patients with RP, which is a disease affects an estimated 82,000-110,000 individuals in the United States.
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 partial tax credits for clinical trial costs, waiver of the user fee for marketing applications and, upon approval, consideration for seven years of marketing exclusivity.
“Retinitis pigmentosa is a serious and incurable sight-threatening disease that represents a major unmet need in the field of ophthalmology,” stated Todd C. Brady, M.D., Ph.D., 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.”
Shares of ALDX are up over 6% in the pre-market session Wednesday. The stock has surged over 37% so far this year.