Drugs

FDA Highlights Rare Disease Day 2023 with ARC

CDER associate director for rare diseases Kerry Jo Lee says FDA is marking Rare Disease Day 2023 by exploring ways to engage and collaborate with patients, patient advocates, and other stakeholders to support the development of safe and effective therapies. Writing in an agency statement, Lee describes the Accelerate Rare disease Cures (ARC) program that was launched in 5/2022.

“The ARC program seeks to harness CDER’s collective expertise and activities to drive scientific and regulatory innovation for rare diseases,” she writes. “It also builds upon CDER’s existing capabilities to expand its interactions with the rare disease stakeholder community. The vision of the ARC program is speeding and increasing the development of effective and safe treatment options to address the unmet needs of patients with rare diseases.”

In its first year, Lee says, the ARC program has focused on strengthening partnerships with patients, advocacy groups, industry, and other stakeholders. Moving forward, she adds, it has multiple efforts underway to support rare disease drug development.

Lee’s paper also covers new drug approvals for rare diseases and Critical Path Institute partnerships.

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