Issuance of Priority Review Voucher for LOARGYS in Rare Pediatric Disease
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The Food and Drug Administration (FDA) has announced the issuance of a priority review voucher to Immedica Pharma AB for their product LOARGYS (pegzilarginase-nbln), which has been approved for the treatment of hyperargininemia in patients with Arginase 1 Deficiency. This law reinforces the FDA's commitment to facilitating expedited review processes for drugs addressing rare pediatric diseases. The issuance is part of the Rare Pediatric Disease Priority Review Voucher Program authorized by the Federal Food, Drug, and Cosmetic Act.
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Key Changes
- Issuance of priority review voucher for the product LOARGYS.
- Recognition of LOARGYS as a treatment for hyperargininemia in pediatric patients.
- Compliance with criteria set out in section 529 of the FD&C Act.
Obligations
What this law requires
FDA must publish notice of the award of priority review vouchers for approved rare pediatric disease products
Sponsors of rare pediatric disease product applications must meet specified criteria established under section 529 of the FD&C Act (21 U.S.C. 360ff) to qualify for a priority review voucher
FDA must determine whether approved rare pediatric disease products meet the criteria for priority review voucher eligibility under section 529 of the FD&C Act
LOARGYS (pegzilarginase-nbln) must be indicated and labeled for treatment of hyperargininemia in adult and pediatric patients 2 years of age and older with Arginase 1 Deficiency in conjunction with dietary protein restriction