FDA Awards Priority Voucher for Rare Kids' Disease Drug Gomekli
Published Date: 3/13/2025
Notice
Summary
The FDA just gave SpringWorks Therapeutics a special priority review voucher for their drug Gomekli, which treats a rare disease in kids and adults with neurofibromatosis type 1. This voucher speeds up future drug reviews, helping get important medicines to patients faster. The approval happened on February 11, 2025, marking a big win for rare disease treatments and innovation.
Analyzed Economic Effects
2 provisions identified: 2 benefits, 0 costs, 0 mixed.
Gomekli approved for NF1 patients
GOMEKLI (mirdametinib) was approved on February 11, 2025. It is indicated for adults and pediatric patients 2 years of age and older with neurofibromatosis type 1 who have symptomatic plexiform neurofibromas, expanding the set of approved treatment options for that patient group.
Priority review voucher awarded to sponsor
The FDA announced issuance of a priority review voucher under section 529 of the FD&C Act to the sponsor of the approved rare pediatric disease product, SpringWorks Therapeutics, Inc.; the notice is dated March 7, 2025 and references GOMEKLI's approval on February 11, 2025. The voucher award is formally published by FDA in this Federal Register notice.
Personalized for You
How does this regulation affect your finances?
Personalize government policy and PRIA will tell you what this federal register document means for your household, plus every other regulation we track. PRIA reads each provision against your financial profile to show you exactly what matters to your wallet.
Key Dates
Department and Agencies
Related Federal Register Documents
2026-16296, Substances Generally Recognized as Safe
The Food and Drug Administration (FDA or we) is proposing to require the submission of generally recognized as safe (GRAS) notices for the use of a human or animal food substance purported to be GRAS under the conditions of its intended use under the Federal Food, Drug, and Cosmetic Act (FD&C Act).
2026-14073, Drug Establishment Registration and Drug Listing Requirements for Establishments Engaged in Distributed Manufacturing and Certain Foreign Establishments
The FDA wants to make it easier for drug makers who work in several locations to register as one company instead of many. They’re also updating rules for foreign drug companies to match new laws about handling health threats. If you’re in drug manufacturing, get ready to comment by September 11, 2026, and keep an eye on possible paperwork changes that could affect your business.
2026-13047, Establishment Registration and Product Listing for Tobacco Products
The FDA wants to make sure all tobacco product makers, both in the U.S. and abroad, register their businesses and list their products. This new rule closes a big gap by including foreign companies, helping the FDA keep better track of tobacco products for public health. Comments on this proposal are open until September 14, 2026, so affected businesses should get ready to update their info and possibly face new costs.
2025-04978, Nonprescription Drug Product With an Additional Condition for Nonprescription Use
The FDA is delaying a new rule about nonprescription drugs that have extra conditions for use. Originally set to start on March 21, 2025, the rule’s start date is now pushed back to May 27, 2025, to allow more time for review. This affects drug makers and consumers by giving everyone extra time to prepare for the new requirements without any immediate cost changes.
2025-01840, Nonprescription Drug Product With an Additional Condition for Nonprescription Use
The FDA’s new rule about nonprescription drugs needing extra conditions before use was set to start on January 27, 2025, but it’s now delayed until March 21, 2025. This delay follows a presidential order to pause and review new rules before they take effect. Drug makers, sellers, and consumers should note the new start date and get ready for the updated rules soon.
2026-20063, Request for Nominations for Voting Members of Food and Drug Administration Advisory Committees
The FDA is looking for smart, qualified people to join its advisory committees and help make important decisions about food and drugs. If you have the right skills, you can apply anytime, but get your nomination in by November 30, 2026, for the best chance at current openings. This is a great chance to influence public health without any cost to you!
Previous / Next Documents
Previous: 2025-04020, Determination That RIOMET (Metformin Hydrochloride) Oral Solution, 500 Milligrams/5 Milliliters Has Not Been Withdrawn From Sale for Reasons of Safety or Effectiveness
The FDA has confirmed that RIOMET (metformin hydrochloride) oral solution hasn’t been taken off the market because of safety or effectiveness problems. This means generic versions can keep getting approved as long as they meet the rules. Patients and pharmacies can keep counting on this medicine without any interruptions or extra costs.
Next: 2025-04022, Change in Bank Control Notices; Acquisitions of Shares of a Bank or Bank Holding Company
Some folks want to buy shares in banks or bank companies, and the Federal Reserve is checking their applications to make sure everything’s fair and safe. If you want to share your thoughts, you’ve got until March 28, 2025, to speak up! This process helps keep banks strong and protects your money.