Biologics

Bryan Concerns About Gene Therapy Accelerated OKs

CBER Office of Tissues and Advanced Therapies director Wilson Bryan, who has announced his retirement from the agency, is concerned about accelerated approvals for cell and gene therapies considering how long it can take to complete confirmatory trials, according to a Bloomberg Law report. Speaking 2/13 at the Biopharma Congress, Bryan said, “If it takes years as suggested to tell if something works, then that means it takes years to tell if it doesn’t work…. I really worry that will damage the field by approving something under accelerated approval that at the end of the day doesn’t work.”

Responding to Bryan’s concern, Yale School of Medicine family physician Reshma Ramachandran told Bloomberg Law that the comments “highlight the challenge that FDA faces in their role balancing timely access to new products, especially where there are no options available for patients, while also providing assurance to patients and clinicians that the product is safe and effective.”

National Organization for Rare Disorders director of policy and regulatory affairs Karin Hoelzer told Bloomberg that accelerated approval has benefited rare disease patients. “We do not want the standards to be so stringent that our patients cannot access gene therapies that have been shown to be effective based on the accelerated approval standard — surrogate or intermediate endpoints that are reasonably likely to predict clinical benefit,” she said.

“As accelerated approval provides a pathway for withdrawing the treatment should it fail confirmatory studies or be unnecessarily delayed in doing so,” Ramachandran said, “it’s critical that FDA has continued oversight as the study goes on, not just many years later when the submission deadline occurs. If there is a safety concern that emerges along the way, FDA should have the ability to stop market authorization of the drug and investigate in a timely manner to prevent further patient harm. Additionally, if there does not seem to be any signal of impending clinical benefit as the study continues, then the study should be stopped earlier and early withdrawal be considered.”

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