Drugs

Funding Bill Reinstates FDA Pediatric Drug Voucher Program

President Trump has signed a government spending bill that officially reinstates FDA’s Rare Pediatric Disease Priority Review Voucher (PRV) program, reviving a key incentive for rare childhood disease drug development that had lapsed more than a year ago. The provision, part of the Consolidated Appropriations Act of 2026 signed on 2/3, restores the program under the Mikaela Naylon Give Kids a Chance Act and extends the PRV program through 9/30/2029.

Under the program, drug sponsors that secure FDA approval for treatments targeting rare pediatric diseases receive a priority review voucher that can be redeemed to shorten FDA review timelines or sold to another company for substantial revenue.

The program had expired 12/2024 after Congress failed to renew it, leaving many rare disease drug developers in regulatory and financial limbo and threatening to stall innovation in treatments for conditions that primarily affect children. With its lapse, only drugs designated before the 2024 deadline had remained eligible for vouchers, and future awards were set to end by 9/30.

Industry groups and patient advocates have broadly championed the PRV program as a successful tool for spurring investment in therapies for rare pediatric conditions, such as Duchenne muscular dystrophy and hemophilia A. Since its creation in 2012, the program has yielded more than 60 vouchers across dozens of diseases, according to advocacy data.

Gupta Raises Concerns About Overton’s Independence, Ideology

FDA commissioner nominee Heidi Overton’s responses on vaccines, contraception and abortion medication during her 9/24 Senate confirmation hearing raise concerns that ideology could influence her decision-making if she takes over the agency, MSNBC senior medical analyst Dr. Vin Gupta said Friday. Gupta was particularly critical of Overton’

Overton Confirmation Remains Uncertain After Senate Hearing

FDA commissioner nominee Heidi Overton emerged from her Senate confirmation hearing Thursday (see earlier story) without resolving questions about whether she has enough Republican support to advance — and with a rapidly narrowing window for confirmation before the midterm elections. The Senate Health, Education, Labor and Pensions Committee hearing provided few

FDA Classifies Boston Scientific Imager II Catheter Recall as Class I

FDA has classified Boston Scientific's recall of its Imager II Angiographic Catheters as Class I because a manufacturing problem could cause catheter tips to degrade and detach, potentially resulting in life-threatening complications. The agency announced the Class I classification 9/25, updating an Early Alert it issued

FDA Approves Gazyva for Childhood-Onset Idiopathic Nephrotic Syndrome

FDA has approved Roche’s Gazyva (obinutuzumab) for reducing the risk of relapse in adults and children aged 2 and older with frequently relapsing or steroid-dependent, childhood-onset idiopathic nephrotic syndrome who are in remission. The expanded approval covers the use of the CD20-directed antibody beyond its existing

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Gupta Raises Concerns About Overton’s Independence, Ideology

Overton Confirmation Remains Uncertain After Senate Hearing

FDA Classifies Boston Scientific Imager II Catheter Recall as Class I

FDA Approves Gazyva for Childhood-Onset Idiopathic Nephrotic Syndrome