2025-06785Notice

AMVUTTRA Gets Priority Nod for Rare Pediatric Conditions via Voucher

Published Date: 4/21/2025

Notice

Summary

The FDA just gave the green light to AMVUTTRA (vutrisiran) for a rare kid’s disease, using a special priority review voucher to speed things up. This means faster access to important medicine for families and shows the FDA’s commitment to helping rare pediatric conditions. The approval happened on March 20, 2025, marking a big win for patients and drug developers alike.

Free Policy Watch

New rules are filed every week. Most people never see them.

Pick a topic. PRIA watches every federal rule and tells you when one hits your household.

Pick a topic to get started

Analyzed Economic Effects

1 provisions identified: 1 benefits, 0 costs, 0 mixed.

FDA Approves AMVUTTRA for Rare Pediatric Disease

If you are a parent or guardian of a child with a rare pediatric disease, the FDA approved AMVUTTRA (vutrisiran) on March 20, 2025. The approval (Supplement-06) redeemed a Rare Pediatric Disease Priority Review Voucher, which the notice says was used for this product's review.

Your PRIA Score

Score Hidden

Personalized for You

How does this regulation affect your finances?

Sign up for a PRIA Policy Scan to see your personalized alignment score for this federal register document and every other regulation we track. We analyze your financial profile against policy provisions to show you exactly what matters to your wallet.

Free to start

Key Dates

Effective Date
Published Date
3/20/2025
4/21/2025

Department and Agencies

Department
Independent Agency
Agency
Health and Human Services Department
Food and Drug Administration
Source: View HTML
Back to Federal Register

Take It Personal

Get Your Personalized Policy View

Start a Free Government Policy Watch to see how policy affects your household, then upgrade to PRIA Full Coverage for year-round monitoring.

Already have an account? Sign in