FDA Awards Fast-Track Voucher for Rare Kids' Blood Disorder Drug
Published Date: 2/23/2026
Notice
Summary
The FDA just gave a special fast-track prize called a Priority Review Voucher to Fondazione Telethon ETS for their rare disease treatment, WASKYRA, approved in December 2025. This medicine helps kids and adults with Wiskott-Aldrich Syndrome who need a stem cell transplant but can’t find a perfect donor. This voucher speeds up future drug reviews and can be sold, saving time and money for drug developers.
Analyzed Economic Effects
2 provisions identified: 2 benefits, 0 costs, 0 mixed.
WASKYRA Approved for WAS Patients
WASKYRA (etuvetidigene autotemcel) was approved on December 9, 2025, for pediatric patients aged 6 months and older and adults with Wiskott-Aldrich Syndrome who have a mutation in the WAS gene and for whom hematopoietic stem cell transplantation (HSCT) is appropriate and no suitable HLA‑matched related stem cell donor is available. If you are in that patient group, this approval makes the treatment an approved option.
FDA Issues Rare Disease Priority Voucher
The FDA issued a Priority Review Voucher to Fondazione Telethon ETS for WASKYRA after its approval on December 9, 2025. The notice says this voucher speeds up future drug reviews and can be sold, which can save time and money for drug developers.
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.
2026-16959, Frequently Asked Questions-Developing Potential Cellular and Gene Therapy Products; Final Guidance for Industry; Availability
The Food and Drug Administration (FDA or Agency) is announcing the availability of a final guidance titled "Frequently asked Questions--Developing Potential Cellular and Gene Therapy Products." The guidance document provides industry with answers to frequently asked questions (FAQs) and commonly faced issues that arise during the development of cellular and gene therapy (CGT) products. The FAQs represent common questions directed to the Agency and span multiple disciplines, including regulatory review; chemistry, manufacturing, and controls (CMC); pharmacology/toxicology; clinical; and clinical pharmacology. This guidance announced in this notice finalizes the draft guidance of the same title issued on November 19, 2024.
2026-16942, Food Additives Permitted in Feed and Drinking Water of Animals; Chromium DL-methionine Chelate
The Food and Drug Administration (FDA, we, or the Agency) is amending the regulations for food additives permitted in feed and drinking water of animals to provide for the safe use of chromium DL- methionine chelate as a nutritional source of chromium in cattle feed. This action is in response to a food additive petition filed by Zinpro Corp.
2026-16939, GNT USA, LLC.; Filing of Color Additive Petition
The Food and Drug Administration (FDA or we) is announcing that we have filed a petition, submitted by GNT USA, LLC., c/o Exponent, Inc., proposing that we amend our color additive regulations to provide for the safe use of safflower (Carthamus tinctorius L.) extract as a color additive in various foods at levels consistent with good manufacturing practices.
Previous / Next Documents
Previous: 2026-03492, Petition for Exemption; Summary of Petition Received; Morningside University
Morningside University has asked the FAA for a special exemption from some aviation rules. This could affect students and staff involved in their aviation programs by changing how certain regulations apply. People have until March 16, 2026, to share their thoughts, and no extra costs or delays are expected right now.
Next: 2026-03495, Determination That Klonopin (Clonazepam) Tablets, 0.125 Milligrams and 0.25 Milligrams, Were Not Withdrawn From Sale for Reasons of Safety or Effectiveness
The FDA has decided that Klonopin tablets in 0.125 mg and 0.25 mg strengths were not taken off the market because of safety or effectiveness problems. This means generic drug makers can now apply to sell their versions of these Klonopin tablets, as long as they meet all other rules. Patients and pharmacies could see more affordable options soon, with no safety worries holding things back.