Issuance of Priority Review Voucher; Rare Pediatric Disease Product; ALHEMO (concizumab-mtci)
Published Date: 1/15/2025
Notice
Summary
The FDA just gave a special fast-track ticket called a Priority Review Voucher to Novo Nordisk for their new rare pediatric disease drug, ALHEMO, approved in December 2024. This voucher helps speed up the review of future medicines, benefiting patients with rare diseases and encouraging companies to develop more treatments. This move affects drug makers and could speed up new medicine approvals, saving time and possibly money.
Analyzed Economic Effects
2 provisions identified: 2 benefits, 0 costs, 0 mixed.
ALHEMO Approved for Hemophilia Prophylaxis
ALHEMO (concizumab-mtci) was approved on December 20, 2024 for routine prophylaxis to prevent or reduce bleeding episodes in adult and pediatric patients 12 years of age and older with hemophilia A with FVIII inhibitors and hemophilia B with FIX inhibitors. If you or a child in your care is in those groups, ALHEMO is an FDA‑approved treatment option as of that date.
Priority Review Voucher Issued to Sponsor
The FDA issued a Priority Review Voucher under section 529 of the FD&C Act to the sponsor of the ALHEMO application (manufactured by Novo Nordisk, Inc.). The notice states ALHEMO met the criteria for a rare pediatric disease priority review voucher following its approval on December 20, 2024.
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