Drugs

26 Rare Disease Drug Approvals in 2024: ARC Report

The FDA Accelerating Rare Disease Cures (ARC) program Year-Three Annual Report says there were 26 rare disease new drug approvals in calendar year 2024. That’s three fewer approvals than ARC reported in its Year-Two report for 2023. Other categories shown on the report’s Year-At-A-Glance page (public speaking engagements, externally-led patient-focused drug development meetings, patient listening sessions, quarterly newsletters, and total number of posts) were either even or lower, except for an increase in the number of subscribers to CDER’s Rare Disease News.

In the report’s look to the future, associate director for rare diseases Scott Winiecki says ARC “expanded efforts in translational medicine approaches for individual rare diseases through efforts such as the Translational Science Team and contributed to the clinical and regulatory knowledge that is needed to make informed decisions for rare disease drug development, as evidenced by multiple publications and research projects….”

Winiecki says that as ARC moves into 2026, it will continue its commitment to engagement and collaboration, fostering resilience in rare disease drug development through innovation and 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