Gene modifying therapy gets US approval

Novartis AG on Wednesday won highly anticipated US approval for the first of a new type of potent gene-modifying immunotherapy for leukemia, a $475,000 treatment that marks the start of a potential new treatment paradigm for some cancers.

The approval was widely expected after an FDA advisory panel last month unanimously recommended the action. Novartis also announced an agreement with the US Centres for Medicare and Medicaid Services under which payment for the therapy will be based on clinical outcomes achieved.

The treatment, called Kymriah, was approved for patients up to 25 years of age who have relapsed or not helped by prior treatment for B-cell acute lymphoblastic leukemia (ALL).

Kymriah belongs to a new class of treatments called CAR-T therapies. It involves removing disease-fighting T cells from a patient, genetically modifying them to better recognize and attack cancer, and then replacing them, where they can circulate for years seeking out the disease.

“Two years ago many people would have told you these types of treatments were science fiction,” said Brad Loncar, chief executive of Loncar Investments, which runs the Loncar Cancer Immunotherapy ETF “We’ve never seen anything like this before and I believe this therapy may become the new standard of care for this patient population,” Dr Stephan Grupp of Children’s Hospital of Philadelphia said in a statement.

However, this type of therapy carries risk of severe side effects. Kymriah will have a boxed warning for cytokine release syndrome, a potentially lethal systemic response to the activation and proliferation of CAR-T cells, causing high fever and potential for neurological problems.