Drugs

Stakeholders Seek Changes in CAR-T Draft Guidance

Pharmaceutical Research and Manufacturers of America (PhRMA) says it believes the recommendations in an FDA draft guidance on considerations for developing chimeric antigen receptor (CAR) T cell products “are essential in helping sponsors navigate development issues in this rapidly evolving field.” In a 6/14 comment letter, PhRMA says that in recognition of the complexity and quickly evolving use of this technology, FDA should periodically assess various technologies related to CAR-T development and update the guidance accordingly. PhRMA also lists other guidance topic areas where it says additional clarifications would be helpful.

The International Society for Pharmaceutical Engineering recommends that FDA add (1) a reference to quality by design, (2) additional visual representations/diagrams for CAR-T construct, (3) patient population demographic considerations, (4) limitations of CAR-T cell therapy, (5) key considerations for setting and determining appropriate trial endpoints, and (6) quality considerations for pre-clinical through late-stage development.

In its letter, Novartis says sponsors must engage with FDA early and often to ensure they are aligned on how best to develop the new therapies. “Current meeting types, including PDUFA meetings, are not an efficient means to seek FDA feedback,” the company says. “Communication opportunities outside of PDUFA meetings are needed.”

Novartis also suggests changes and clarifications for sections on chemistry, manufacturing, and controls (CMC); non-clinical studies; clinical studies; and long-term follow-up.

AstraZeneca recommends changes in three sections of the draft — CMC; pre-clinical; and clinical dosing.

Finally, Gilead’s Kite subsidiary says FDA should consider providing more specific guidance on the stage-appropriate CMC information for submission, including examples.

All five stakeholders also provided line-by-line specific comments.

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