The Food and Drug Administration (FDA) has issued a priority review voucher to PTC Therapeutics Inc. for their product, KEBILIDI (eladocagene exuparvovec-tneq). This product is designed to treat both adult and pediatric patients with a condition known as aromatic L-amino acid decarboxylase deficiency. The priority review voucher is awarded under the Rare Pediatric Disease program, which encourages the development of treatments for rare diseases affecting children. The FDA is required to announce when such vouchers are given, as part of the criteria set by the Federal Food, Drug, and Cosmetic Act.
Simple Explanation
The FDA has given a special reward, like a golden ticket, to a company for making a new medicine that helps kids with a rare sickness. This reward helps the company get faster help to get their medicine ready for everyone who needs it.