Biologics

Guide on Studying Multiple Cell/Gene Therapies

FDA has released a final guidance entitled “Studying Multiple Versions of a Cellular or Gene Therapy Product in an Early-Phase Clinical Trial.” The document provides recommendations to sponsors interested in studying (umbrella trials) multiple versions of a cellular or gene therapy product in an early-phase clinical trial for a single disease. It also discusses recommendations on how to organize and structure the INDs, submit new information, and report adverse events. “Although multiple versions of a product can be studied together in a single clinical trial, each version of the product is distinct and is generally submitted to FDA in a separate investigational new drug application (IND),” it says.

The benefits from running this type of umbrella trial include flexibility and efficiency in product development, the agency says. “Instead of an iterative approach to clinical studies, multiple versions of a cellular or gene therapy product can be studied in parallel, which may streamline early clinical development by expeditiously identifying alternative versions of a product that may be safer or more effective,” the guidance explains. “Such comparisons can be facilitated by randomization between the study arms, if feasible. Additionally, this trial design may facilitate sharing of the control group, potentially facilitating investigator participation and subject enrollment, and may simplify study management, relative to conducting a separate clinical trial for each product version.”

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