Search Results for keywords:"Rare Pediatric Disease"

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Search Results: keywords:"Rare Pediatric Disease"

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  • 2021-03012 — Issuance of Priority Review Voucher; Rare Pediatric Disease Product
    Type:Notice
    Citation:86 FR 9514
    Reading Time:about a minute or two

    The Food and Drug Administration (FDA) has awarded a priority review voucher to Alnylam Pharmaceuticals Inc. for their product, OXLUMO (lumasiran) injection. This product is used to treat a rare disease called primary hyperoxaluria type 1, which helps lower urinary oxalate levels in both children and adults. The voucher is part of a program set by the Federal Food, Drug, and Cosmetic Act, which recognizes drugs designed for rare pediatric diseases. The FDA is required to announce when such vouchers are awarded.

    Simple Explanation

    The FDA gave a special "golden ticket" to a company called Alnylam for making a medicine that helps kids with a very rare sickness. This golden ticket makes it faster for any of their future medicines to get approved more quickly.

  • 2025-00750 — Issuance of Priority Review Voucher; Rare Pediatric Disease Product; ALHEMO (concizumab-mtci)
    Type:Notice
    Citation:90 FR 3880
    Reading Time:about a minute or two

    The Food and Drug Administration (FDA), part of the Department of Health and Human Services, has issued a priority review voucher to Novo Nordisk, Inc. for their drug ALHEMO (concizumab-mtci), which is used to reduce bleeding episodes in patients with certain types of hemophilia. This award is part of the FDA's program to encourage the development of treatments for rare pediatric diseases. The voucher is given because ALHEMO met specific criteria for applications targeting rare pediatric conditions.

    Simple Explanation

    The FDA gave a special prize, called a priority review voucher, to a company named Novo Nordisk because they made a medicine called ALHEMO that helps kids with a rare illness bleed less. This prize encourages the creation of medicines for kids with uncommon diseases.