FDA Approves First Gene Therapy in US

FDA Approves First Gene Therapy in US

The CAR-T cell treatment developed by Novartis and the University of Pennsylvania is the first type of gene therapy to hit the USA market - and one in a powerful but expensive wave of custom-made "living drugs" being tested against blood cancers and some other tumours.

"We are so proud to be part of this historic moment in cancer treatment and are deeply grateful to our researchers, collaborators, and the patients and families who participated in the Kymriah clinical program", added Bruno Strigini, CEO of Novartis Oncology.

About 15 to 20 percent of patients with B-cell ALL have cancer that either did not respond to treatment or has recurred, the FDA says. In June, a clinical trial in China showed promise in treating another blood cancer, multiple myeloma, using reprogrammed T-cells in much the same way Kymriah does.

The new treatment involves removing immune cells called T cells from a patient's blood and giving them a gene for a protein called a chimeric antigen receptor, or auto, that directs the T cells to target leukemia cells.

The FDA said Wednesday it approved Novartis AG's Kymriah for certain children and young adults who suffer from a form of leukemia.

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It's too soon to tell how widely applicable CAR-T will be - it's especially suited to leukemia since infusing patients with T-cells is relatively simple.

"There has been an urgent need for novel treatment options that improve outcomes for patients with relapsed or refractory B-cell precursor ALL", Novartis, the drug company that makes Kymriah, said in a statement. Under the arrangement, Novartis will only collect payments from CMS when patients respond to treatment by the end of the first month.

However, this type of therapy is not without risk of severe side effects as the immune system goes into high gear.

The one-time treatment has a boxed warning for cytokine release syndrome or CRS, a life-threatening side effect that can cause blood pressure to drop dangerously low.

Because of these safety concerns, the FDA will require that hospitals receive special certification to use Kymriah, the agency said. The agency granted approval of the gene therapy to Novartis Pharmaceuticals Corp.

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The first gene therapy in the United States has been approved by the Food and Drug Administration (FDA).

Analysts have speculated the treatment could cost several thousand dollars.

"Although narrow in scope, today's FDA ruling is a milestone", said Dr. David Maloney of the Fred Hutchinson Cancer Research Center in Seattle, whose team has worked with Juno and is researching CAR-T in a variety of cancers.

"The theory is they should attack the tumor and continue to grow to become a long-term monitoring and treatment system", Lichtenfeld said. More than half were children and teens.

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