Drugs

Human Genome Editing Product Development Guide

FDA has issued a guidance entitled “Human Gene Therapy Products Incorporating Human Genome Editing.” The document provides recommendations about the information that should be included in an IND in order to assess the safety and quality of the investigational genome editing (GE) product, such as product design, product manufacturing and testing, nonclinical safety assessment, and clinical trial design. The agency says human GE is a “process by which DNA sequences are added, deleted, altered or replaced at specified location(s) in the genome of human somatic cells, ex vivo or in vivo, using nuclease-dependent or nuclease-independent GE technologies.”

FDA notes that the interest in GE as a scientific technology for treating human diseases has increased substantially, and there has been rapid development of gene therapy products relying on GE. “While the potential of such products for the treatment of human disease is clear, the potential risks are not as well understood,” the guidance says. “To assist in the translation of these products from the bench to clinical trials, this guidance includes recommendations for how to assess the safety and quality of these products and address the potential risks of these products.”

Additionally, the guidance says that some of the specific risks associated with GE approaches include “off-target editing, unintended consequences of on-target editing, and the unknown long-term effects of on- and off-target editing.” Because human GE is a rapidly evolving field, FDA says it may revise its recommendations on developing GE products as they gain information on the safety of such products.

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