Drugs

New Rare Disease Efforts to Help Product Development

In recognition of Rare Disease Day (2/28), FDA has announced new actions to advance development and approval of therapies to such diseases. For example, a new request for applications (RFA) has been posted within the agency’s Orphan Products Grants Program for “Efficient and Innovative Natural History Studies Addressing Unmet Needs in Rare Diseases.” The funding opportunity, FDA says, is intended to support natural history studies with high quality and interpretable data elements that will “address critical knowledge gaps, remove major barriers to progress in the field, exert a significant and broad impact on a specific rare disease or multiple rare diseases with similar pathophysiology, and facilitate rare disease product development.”

Additionally, CBER is working to advance the development of individualized therapies for rare diseases that affect one or a few individuals. “Stakeholders recognize the need to have an end-to-end approach for development of individualized (bespoke) gene therapies and sustainable access for patient populations that can benefit from these therapies,” FDA says. The Center is collaborating with the Foundation for the National Institutes of Health and the National Center for Advancing Translational Sciences to create a “Bespoke Gene Therapy Consortium” to provide a standardized and efficient approach for developing bespoke Adeno-associated virus-based gene therapies. Efforts, it says, include collaborative solutions to address current challenges and regulatory innovations to streamline the development process.

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