2025-04021Notice

FDA Hands Out Speedy Voucher for Kids' Rare Disease Breakthrough

Published Date: 3/13/2025

Notice

Summary

The FDA just gave SpringWorks Therapeutics a special priority review voucher for their rare pediatric disease drug, Gomekli (mirdametinib), approved on February 11, 2025. This voucher speeds up the review of future drug applications, helping get important medicines to kids faster. Companies can use or sell these vouchers, which can save time and money in drug approval processes.

Analyzed Economic Effects

2 provisions identified: 2 benefits, 0 costs, 0 mixed.

Company awarded tradable priority voucher

The FDA issued a priority review voucher to SpringWorks Therapeutics for GOMEKLI (mirdametinib) after the drug's approval on February 11, 2025. The voucher can be used or sold to speed the review of a future drug application, giving the company a tradable asset that can save time and money in the approval process.

Faster access to pediatric drugs

Priority review vouchers let companies speed the review of future drug applications, which can help get important medicines to children with rare diseases faster. Because companies can use or sell the voucher, this may lower time and money barriers to bringing pediatric treatments to market.

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Key Dates

Effective Date
Published Date
2/11/2025
3/13/2025

Department and Agencies

Department
Independent Agency
Agency
Health and Human Services Department
Food and Drug Administration
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