The Food and Drug Administration (FDA) has announced the approval of a product that uses a priority review voucher under the Rare Pediatric Disease Priority Review Voucher Program. This program, authorized by the Federal Food, Drug, and Cosmetic Act, allows the FDA to award vouchers to sponsors of approved drug applications for rare pediatric diseases. The supplemental application for AMVUTTRA (vutrisiran), approved on March 20, 2025, met the criteria for redeeming such a voucher, demonstrating its commitment to addressing rare diseases in children.
Simple Explanation
The FDA gave a special permission ticket to help a medicine for kids, called AMVUTTRA, get reviewed faster and be ready for children who need it. This ticket is part of a program to encourage making medicines for rare kid diseases.