The Priority Review

Tag

Pediatrics

3 stories

  1. Approvals

    FDA approves first pediatric heart valve built to be expanded as a child grows

    Edwards Lifesciences' Autus Size-Adjustable Valve is also the first valve approved in the U.S. with polymeric leaflets instead of animal tissue.

  2. Approvals

    FDA approves Emcitate, the first treatment for MCT8 deficiency

    Egetis Therapeutics' tiratricol treats the excess blood thyroid hormone of Allan-Herndon-Dudley syndrome. The company also received a rare pediatric disease priority review voucher.

  3. Approvals

    FDA approves Fayuvi, the first gene therapy for Sanfilippo syndrome type A

    Ultragenyx's one-time AAV9 infusion received standard full approval for children with MPS IIIA, and the company received a priority review voucher.