Search Results for keywords:"rare pediatric disease"

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Search Results: keywords:"rare pediatric disease"

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  • 2024-28206 — Issuance of Priority Review Voucher; Rare Pediatric Disease Product; KEBILIDI (eladocagene exuparvovec-tneq)
    Type:Notice
    Citation:89 FR 95219
    Reading Time:about a minute or two

    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.

  • 2025-00340 — Issuance of Priority Review Voucher; Rare Pediatric Disease Product; CRENESSITY (crinecerfont)
    Type:Notice
    Citation:90 FR 2008
    Reading Time:about a minute or two

    The Food and Drug Administration (FDA) has issued a priority review voucher to the sponsor of the rare pediatric disease product, CRENESSITY (crinecerfont). This voucher is awarded under the Federal Food, Drug, and Cosmetic Act, which allows such recognition for approved products meeting specific criteria. CRENESSITY is manufactured by Neurocrine Biosciences, Inc., and was approved on December 13, 2024, for the treatment of classic congenital adrenal hyperplasia in adults and children aged four and older. This announcement fulfills FDA's requirement to publish notice of such awards.

    Simple Explanation

    The FDA gave a special ticket to a medicine called CRENESSITY that helps children with a rare illness so it could be checked faster, and they had to tell everyone they did this.

  • 2025-00341 — Notice of Approval of Product Under Voucher: Rare Pediatric Disease Priority Review Voucher; ALYFTREK (vanzacaftor, tezacaftor, and deutivacaftor)
    Type:Notice
    Citation:90 FR 2006
    Reading Time:about a minute or two

    The Food and Drug Administration (FDA) has announced that they approved the use of ALYFTREK, a drug made up of vanzacaftor, tezacaftor, and deutivacaftor, on December 20, 2024. This approval includes the use of a priority review voucher, which is a special designation given to sponsors of drugs for rare pediatric diseases. These vouchers are part of a program designed to encourage the development of treatments for rare conditions found in children. For more information, individuals can visit the FDA's official website linked in the document.

    Simple Explanation

    The FDA has said "yes" to a new medicine called ALYFTREK that helps kids with rare illnesses, and they're using a special ticket to get it approved faster. Think of the ticket as a "skip the line" pass at a theme park to help the medicine get to kids who need it more quickly.

  • 2025-04021 — Issuance of Priority Review Voucher; Rare Pediatric Disease Product; Gomekli (mirdametinib)
    Type:Notice
    Citation:90 FR 11992
    Reading Time:about a minute or two

    The Food and Drug Administration (FDA) has announced the issuance of a priority review voucher to SpringWorks Therapeutics, Inc. for their drug GOMEKLI (mirdametinib). This drug is used to treat adults and children 2 years and older who have neurofibromatosis type 1 with symptomatic plexiform neurofibromas. The priority review voucher is awarded under the Federal Food, Drug, and Cosmetic Act to incentivize companies to develop treatments for rare pediatric diseases. The notice was officially filed on March 12, 2025.

    Simple Explanation

    The FDA is giving a special prize to a company for making a new medicine called Gomekli, which helps kids and grown-ups with a rare health problem. This prize makes it quicker and easier for medicines to be checked and used.