Search Results for keywords:"section 529 FD

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.

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 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), 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.

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.

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) is updating animal drug regulations to reflect actions related to new animal drug applications (NADAs) and abbreviated new animal drug applications (ANADAs) from October to December 2024. Some approvals for specific animal drugs have been withdrawn because they are no longer manufactured or marketed. Additionally, the FDA is making technical amendments to enhance the accuracy and clarity of the regulations, updating sponsor information, and making adjustments in drug sponsorships and addresses. These changes take effect on January 21, 2025.

The Securities and Exchange Commission (SEC) is asking for comments on the information collection related to Regulation FD, which requires public companies to share important information with all investors equally. This regulation aims to prevent selective disclosure, ensuring that material information is available to everyone at the same time. The SEC estimates that roughly 7,196 companies each make five such disclosures annually, resulting in 19,274 responses and about 96,370 total hours of effort. They are seeking feedback by March 31, 2025, on whether the collection is useful, if their time estimates are correct, and how the process might be improved or made less burdensome.