Search Results for keywords:"rare pediatric disease"

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.

The Food and Drug Administration (FDA) has announced the approval of a product called TREMFYA (guselkumab) using a priority review voucher. This type of voucher is given by the FDA under the Federal Food, Drug, and Cosmetic Act to sponsors with approved treatments for rare pediatric diseases. The approval of TREMFYA, which was finalized on March 20, 2025, met all necessary criteria for using the voucher. Information on both the priority review voucher program and TREMFYA is available on the FDA's website.

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.

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.

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.

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.