The Priority Review

Tag

Rare disease

2 stories

  1. 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.

  2. 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.