Drugs

Nature Editorial Urges Academic Scientists to Help FDA

As advancing science is fueling new discoveries, FDA needs help from academic scientists to review and approve new drug therapies because in-house talent is weak, according to a Nature editorial. To illustrate the challenges, the editorial points to a 7/12 FDA advisory committee panel that reviewed a Novartis BLA for CTL019 (tisagenlecleucel-T), a chimeric antigen receptor T-cell (CAR-T) for treating pediatric and young adult patients with relapsed or refractory B-cell acute lymphoblastic leukemia. CAR-T therapies are different from traditional drug or biologic therapies because they are manufactured for each individual patient using their own cells. “During the treatment process, T cells are drawn from a patient's blood and reprogrammed in the manufacturing facility to create T cells that are genetically coded to express a chimeric antigen receptor to recognize and fight cancer cells and other B-cells expressing a specific antigen,” according to the company.

While traditional advisory committee meetings review safety and efficacy data and debate statistical details and clinical-trial design, the Novartis BLA panel spent half of the meeting discussing how the therapy is manufactured, the editorial says. “This example offers a glimpse of the regulatory complexity that cutting-edge therapies present,” it says. “When a treatment is based on a person’s own cells, how can the manufacturer ensure that each dose is as safe and potent as the last? Or when a treatment involves a virus, how can a company ensure that it will be safe over the long term, will not develop the ability to replicate and will not, itself, cause cancer?”

As FDA is forced to delve into new science, “a continuing dearth of expertise will become only more painful,” the editorial notes. “Some upcoming therapies that will land on FDA desks touch on the hottest fields in biomedical research. At the 7/12 meeting, researchers could not hold back from listing how future research could optimize more therapies. Scientists should offer the FDA and other regulators the help to assess them.”

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