TGA approves first CAR-T therapy

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Another milestone in Australian patient access to the first CAR-T therapy with the TGA approving Novartis' KYMRIAH (tisagenlecleucel).

The regulator approved KYMRIAH for the treatment of paediatric and young adult patients up to 25 years of age with B-cell precursor acute lymphoblastic leukaemia (ALL) that is refractory, in relapse post-transplant, or in second or later relapse. It was also approved for the treatment of adult patients with relapsed or refractory diffuse large B cell lymphoma (DLBCL) after two or more lines of systemic therapy. 

KYMRIAH is the first in a new generation of therapies that, unlike small molecules or biologicals, are manufactured for each individual patient using their own cells.

During the treatment process, T cells are drawn from a patient’s blood and reprogrammed in the laboratory to create T cells that are genetically coded to recognise and fight the patient’s cancer cells and other B cells expressing a particular antigen.

"The TGA approval of KYMRIAH is a defining moment for patients in Australia with these aggressive blood cancers," said Lauren Carey, general manager oncology and country president of Novartis Australia and New Zealand.

"KYMRIAH is a one-time treatment that helps address significant treatment gaps and a profound unmet need for patients.”

KYMRIAH has been considered by the Medical Services Advisory Council for funding through the Medical Benefits Schedule.

Health minister Greg Hunt raised the issue of a funding framework for CAR-T therapies with his state and territory counterparts at a recent health COAG meeting. He proposed they be supplied, dispensed and administered through public hospitals.

ALL is a type of cancer that affects the blood and bone marrow. In Australia, it is the most common type of childhood leukaemia, and the outlook is poor for patients who relapse from standard of care therapies. These poor outcomes occur in spite of patients having to undergo multiple treatments, including chemotherapy, radiation, targeted therapy or stem cell transplant.

DLBCL is an aggressive, complex and difficult to treat form of non-Hodgkin lymphoma, accounting for up to 40 per cent of all cases globally. There are limited treatment options that provide durable responses for patients who relapse or do not respond to initial therapy. Survival rates are low for patients who are ineligible for autologous stem cell transplant (ASCT) or because salvage chemotherapy or ASCT have failed.

"Australia is one of the first places in the world to issue a regulatory approval of KYMRIAH, after the United States and Europe," said Richard Vines, founder and chairman of Rare Cancers Australia. "We have seen a number of Australians travelling to the United States for this treatment and today’s announcement gives hope to patients and their families who are running out of treatment options."

"We are entering a transformative time in cancer care. Novartis is delighted about today’s announcement and the meaningful difference this therapy will make to patients in Australia," added Ms Carey. "The next step is to ensure public funding for KYMRIAH so it is accessible for all eligible patients and we are committed to working with the Australian Government to achieve this as soon as possible."