Drugs

Individualized Antisense Oligonucleotide IND Guidance

FDA has published a draft guidance, IND Submissions for Individualized Antisense Oligonucleotide Drug Products: Administrative and Procedural Recommendations, to help sponsor-investigators develop such drug products for a severely debilitating or life-threatening genetic disease. The document focuses on the administrative and procedural aspects of interacting with FDA on development programs for individualized antisense oligonucleotide (ASO) drug products, such as the approach to obtaining feedback from the agency and the expectation and process for making regulatory submissions to FDA.

The guidance provides high-level recommendations about informed consent and the requirement for institutional review board review of protocols for trials of individualized ASO products, FDA says. It also addresses the initial development of the products but does not address regulatory considerations for the development of the drug products for marketing and continued, long-term treatment of patients with the disease for which the drug product is being developed. And the guidance does not address the nonclinical data, clinical data, or the product quality requirements that must be met to initiate administration of the individualized ASO products in humans.

“Although the guidance is intended to help sponsors seeking to develop an individualized ASO drug product,” the document says, “the principles and practices outlined in the guidance may be applicable to developing other types of individualized drug products (i.e., non-ASO). Sponsors who consider applying the principles outlined in this guidance for non-ASO individualized drug products should first consult with the appropriate review division.”

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