Drugs

House OKs Rare Pediatric Disease Voucher Program

The House has approved the Mikaela Naylon Give Kids a Chance Act, which reauthorizes FDA’s Rare Pediatric Disease Priority Review Voucher program. House Energy and Commerce Committee chairman Brett Guthrie (R-KY) says in a committee statement that the bill “builds on current programs to accelerate research and drug development for rare pediatric diseases, including cancer.” The bill was named in honor of Mikaela Naylon, a young woman who courageously advocated for its passage throughout her fight with cancer.

The statement gives this background on the bipartisan legislation:

reauthorizes the Rare Pediatric Disease Priority Review Voucher program through FY 2029 and clarifies that orphan drug exclusivity applies to the approved indication, rather than the potentially broader designation;
gives FDA additional authority to require pediatric cancer trials for new combinations of drug therapies;
authorizes the agency to take enforcement action against companies that fail to meet pediatric study requirements under the Pediatric Research Equity Act;
directs FDA to establish an office in an Abraham Accord country (Israel, the United Arab Emirates, Bahrain, or Morocco) to enhance facilitation with the agency; and
requires FDA to disclose to certain generic drug applicants if any ingredients cause a drug to be quantitatively and qualitatively different from the listed drug, speeding patient access to more affordable medicines.

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