Cellectis: FDA Gives Orphan Drug, Rare Pediatric Status to Leukemia Treatment

By Mark R. Long

Cellectis said the U.S. Food and Drug Administration had granted orphan drug and rare pediatric disease designations to the French biopharmaceutical company's candidate for treating acute lymphoblastic leukemia, or ALL.

The FDA grants orphan drug status to treatments for diseases that affect fewer than 200,000 people. Cellectis said the designation could help speed and cut the costs of developing and getting approval for its UCART22 drug candidate. Rare pediatric disease status could lead to a priority review voucher, the company added.

Cellectis said there was an urgent need to develop treatments for patients who aren't candidates for other therapies. About 10% of the leukemia cases in the U.S. are ALL, and about 1,560 deaths in the U.S. were related to the disease in 2022.

The company said clinical data presented in December were encouraging, and that it expects to give updates on the progress of a study of UCART22 by year's end. UCART22 is a CAR T-cell therapy, a form of immunotherapy that genetically alters immune cells to find and destroy cancer cells.

Write to Mark R. Long at mark.long@wsj.com

(END) Dow Jones Newswires

July 25, 2024 17:53 ET (21:53 GMT)

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