First Gene Therapy treatment approved for release by FDA

Posted May 9, 2017

Novartis approved to market KYMRIAH in the US

Novartis have been approved to market their new cancer Gene Therapy treatment (KYMRIAH) in the United States of America by the US FDA. This marks a significant step forward for the Gene therapy market, as this is the first Gene or Cell Therapy treatment approved by the US FDA. There has been a lot of activity in the pharmaceutical market over the last 10 years, not just in therapy development, but also with new processes and technologies being developed to support the complex nature of delivery.

Today while the frontiers of Biotech engineering and research are still being explored and understood we are entering a new exciting phase as we begin to develop treatments in cell and Gene therapy. And similar to the significant step up in complexity in the production and delivery from Small Molecule Pharma to Large molecule Biotech, we are seeing another step up again as we move into Cell and Gene Therapy.

This step up in complexity is reflected in the purchase price of the drug, which appears to be very high when you compare it to conventional drugs. This doesn’t take into account the complexity and uniqueness of the therapy and it’s delivery. The following extract taken from the FDA press release on 30th August 2017, explains some of the reasons for the treatment complexity and the associated price. Click here for the the article.

“Kymriah is a genetically-modified autologous T-cell immunotherapy. Each dose of Kymriah is a customized treatment created using an individual patient’s own T-cells, a type of white blood cell known as a lymphocyte. 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 leukaemia cells that have a specific antigen (CD19) on the surface. Once the cells are modified, they are infused back into the patient to kill the cancer cells.”

Kymriah is approved as the treatment of last resort once other treatments have been tried and exhausted. This has led to unique pressures in the process. When Novartis were doing clinical trials on this treatment they were finding that the six week laboratory lead-time to take the patients T-cells and genetically modify them was too long. Many patients were not surviving long enough to receive treatment. Today the Novartis labs return the modified patient T-cells in approx. three weeks.

As more complex Cell and Gene therapy treatments are approved by regulators, we need to understand what this means for our healthcare systems and the new skills and increased costs that result from this. This is only the beginning of a conversation which we all need to be part of!