Drugs

FDA Opening Rare Disease Innovation Hub

CDER director Patrizia Cavazzoni and CBER director Peter Marks say they will co-lead a new Rare Disease Innovation Hub (the Hub) to work across rare diseases, but to especially focus on products intended for smaller populations or for diseases where the natural history is variable and not fully understood. An FDA Voices post from Cavazzoni and Marks says the Hub will have three primary functions:

be a single point of connection and engagement with the rare disease community;
enhance inter Center collaboration to address common scientific, clinical, and policy issues related to rare disease product development; and
advance regulatory science with dedicated workstreams for consideration of novel endpoints, biomarker development and assays, innovative trial design, real-world evidence, and statistical methods.

The post says the Hub will leverage activities of the CDER Accelerating Rare Disease Cures program and the CBER Rare Disease Program and will enhance existing cross-Center collaborations. A newly-created position of Director of Strategic Coalitions for the Hub will be a single point of connection and engagement with outside parties on behalf of the Hub on cross-cutting rare disease-related issues. The director also will develop approaches to ensure appropriate FDA staff involvement or appropriate settings for further external engagement and will seek input from the community to inform the Hub’s priorities, the notice says.

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

Read more

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