Gene Therapy Approval for Cancer Treatment is First of Its Kind

Despite these negatives, FDA approval of gene therapy is a big step for the treatment both in the us and around the globe.

Novartis and other companies have been racing to develop gene therapies for other types of cancers, and experts expect more approvals in the near future.

The immunotherapy now can be used in children and young adults with B-cell ALL that will not respond to other therapies, the FDA announced. In a clinical trial, the therapy left 83 percent of such patients cancer-free after three months. It kills more than 3,000 children and young adults in the USA each year and 15% relapse from those who survived despite today's best treatments.

The Food and Drug Administration has approved a new type of cancer treatment for children.

"While both external and Novartis' quantitative assessments of these values indicate that a cost-effective price could be $600,000 to $750,000, we recognize the importance of this paradigm-shifting therapy and are setting the price at $475,000 for this one-time treatment", Dana Cooper, a spokesman for Novartis, said in an interview with OncLive.

"We're entering a new frontier in medical innovation with the ability to reprogram a patient's own cells to attack a deadly cancer", said FDA Commissioner Dr. Scott Gottlieb.

CAR-T offers a new treatment approach in that it is specifically manufactured for each individual patient.

CAR-T treatment is different from the popular immunotherapy drugs that treat cancers by helping the body to spot tumors easily.

"The CAR T cells, they find the cancer cells, and the cancer cells can not hide anymore".

Success with those patients is key to Novartis turning Kymriah into the $1 billion-a-year blockbuster drug the Swiss company predicts it will eventually become, but analysts say it is anyone's guess when it will start covering its costs. The FDA approval of this therapy is based on the results of the Phase II ELIANA trial, which was sponsored by Novartis and included 25 centres in the US, EU, Canada, Australia and Japan. The therapy involves harvesting a patient's T-cells and then genetically modifying them to hold potent molecules called chimeric antigen receptors (CARs).

The $475,000 price tag itself tells only part of the story.

The approval of tisagenlecleucel was based on phase II results from the single-arm, global ELIANA trial of 63 patients who received a single dose of tisagenlecleucel.

Kymriah does have side effects, causing cytokine release syndrome, a systemic response to the proliferation of auto T-cells that can cause flu-like symptoms such as a high fever, and neurological events. Indication-specific pricing is an approach pharmacy benefits manager Express Scripts has been pushing for other cancer drugs.

Treatment with Kymriah has the potential to cause severe side effects.

An Australian representative for Novartis Pharmaceuticals, which sponsored the US-approved treatment, would not comment on whether it planned to seek approval from the TGA to make the treatment commercially available in Australia.

  • Delores Daniels