First Gene Therapy Approved in US

Brandon Parsons
August 31, 2017

After three months of treatment, 83 percent of the patients remained cancer-free. "As a breakthrough immunocellular therapy for children and young adults who desperately need new options, Kymriah truly embodies our mission to discover new ways to improve patient outcomes and the way cancer is treated". The patient's T-cells are collected and sent to a manufacturing center where they are genetically modified to include a new gene that contains a specific protein (a chimeric antigen receptor or CAR) that directs the T-cells to target and kill leukemia cells that have a specific antigen (CD19) on the surface.

While most survive, about 15% relapse despite the current best treatments, and their prognosis is bleak.

In its announcement, Novartis said it plans to make additional filings for Kymriah in the USA and EU this year, including applications with the FDA and European Medicines Agency for the treatment of adult patients with r/r diffuse large B-cell lymphoma. The company did say that it's made an agreement with the US government to pay for the drug only when pediatric or young adult patients with the cancer respond to treatment by the end of their first month. The modified cells are then transfused back.

The FDA based its decision on a clinical trial involving 63 patients with B-cell ALL.

However, this type of therapy is not without risk of severe side effects as the immune system goes into high gear. Gilead shares jumped 5.5 percent on Wednesday as Kite is widely seen as likely to receive the next USA approval of a CAR-T therapy. Other possible severe side effects could include serious infection, low blood pressure, kidney injury, fever and decreased oxygen.

The drug Kymriah was developed by Novartis Pharmaceuticals and the University of Pennsylvania. But speaking earlier this year to The New York Times, drug industry analysts estimated that individualized therapies could cost more than $300,000. Clinical trials using CAR-T cells showed 69% of patients had complete resolution of CRS within 2 weeks following 1 or 2 doses of tocilizumab, according to the release.

Other genetic therapies for cancer are also in the research pipeline.

Even before Novartis' Kymriah was approved, there was concern about pricing of the new therapy, given that new therapies are typically priced based on level of innovation and cancer is a particularly expensive area. More than half were children and teens.

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