Biologics

Marks Sees ‘Breakout Year’ in Gene Therapy Approvals

CBER director Peter Marks is predicting that 2024 will be a “breakout year” for gene therapy approvals as several initiatives work to advance the development of such products. Speaking 2/26 at the BioPharma Congress, Marks said, “What we are trying to accomplish this year is taking a number of the developments that have started to accelerate over the past year, then taking what we learn and apply it. That means applying the accelerated approval provision and optimizing communications with sponsors and increasing our global cooperation with other regulators.”

The Regulatory Affairs Professionals Society reports online that the initiatives that are underway include a recently finalized guidance stating that the accelerated approval pathway may be used for sponsors developing gene therapy products that incorporate gene editing of human somatic cells and the Center’s recently announced Support for clinical Trials Advance Rare disease Therapeutics (START) communications pilot for rare diseases.

Marks made these additional points:

the newly-established Office of Therapeutic Products is 75-80% staffed and still recruiting;
there have been expressions of interest in the START pilot but he would have expected more applications;
the recent approvals for CRISPR gene editing for sickle-cell disease are considered to be gene therapies and not medical devices; and
a draft guidance on the platform technology designation program should be out soon.

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