Biologics

Marks OK with ‘Some’ Gene Accelerated Approval Mistakes

CBER director Peter Marks told the American Society of Gene & Cell Therapy conference on 5/8 that if FDA is going to err in granting accelerated approval for a gene therapy product to treat a rare disease, he would rather err on the side of bringing a potentially useful therapy to patients who need it. “I would much rather take the chance that we’re occasionally going to make an error and give something an accelerated approval than have people so desperate that they’re going out and either going overseas to get unproven therapies, which happens, or using other pathways [where] FDA doesn’t have much regulatory oversight,” he declared. His comments were reported online in Biospace.

Interviewed at a conference “fireside chat,” Marks said the agency’s goal is to get accelerated approval right “90% of the time or more.” But he also said, “We’re not keeping score.”

In his other comments, Marks said:

CBER has to consider the entire ecosystem of gene therapies, recognizing that prices such as the $4.25 million projected price Orchard Therapeutics’ Lenmeldy (atidarsagene autotemcel) one-time therapy for metachromatic leukodystrophy will ultimately prevent the field of gene therapy from being able to treat more common diseases on a global scale;
CBER must work to improve factors such as manufacturing to help make novel therapies more widely available;
FDA would only decide to withdraw a gene therapy if it’s “pretty clear” that any benefits do not outweigh the risks;
he loves adeno-associated viruses but there are manufacturing challenges that threaten their growth;
it’s breathtaking how fast CRISPR developments are going;
the new Office of Therapeutic Products super office is doing an excellent job despite being behind in recruiting and hiring staff; and
a new communications pilot was announced in the Federal Register to focus on rare diseases that are imminently life-threatening and neurologic diseases that have serious outcomes.

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