Senior paediatric oncologist Dr Geoff McCowage at The Children's Hospital at Westmead, NSW, said a similar CAR-T cell therapy was being developed in Sydney and hoped to treat patients within the next 12 months. About 3,100 patients who are 20 and younger are diagnosed with ALL each year.
What sets the drug CAR-T apart from others, is that it is known as a "living drug".
Future uses for the drug in more cancers "could be much larger, but the offset to a larger population would be a lower price most likely", Anderson said in a note to clients. "The lessons learned from the clinical trials of auto T-cell operations will assist us in being able to provide to the larger population". The price is well below the $700,000 that was estimated before the approval of the drug.
While patient groups hailed the potent immunotherapy as a potential cancer game-changer, the Swiss drugmaker was also criticized for setting a price that places Kymriah among the most-expensive drugs ever.
Known as CAR-T therapy, the treatment involves extracting a patient's immune system cells and modifying them to attack aggressive blood cancers.
Those arguments didn't assuage David Mitchell, founder of Patients for Affordable Drugs, and Jamie Love, director nonprofit Knowledge Ecology International, who believe the drug should be more affordable given that taxpayers funded early research.
Another group, the Campaign for Sustainable Rx Pricing said in a statement that while the "science behind Kymriah is revolutionary, the business decision to price it at almost half of a million dollars per treatment is not". The cost of the treatment?
That first patient had been near to death, and according to BBC News has now been cancer-free for more than five years. 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.
And while the path to safe and effective gene therapies has been a long one, "Today, a pivotal leg in that journey is complete", Gottlieb said.
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. They'll also have to be less toxic, so more people can consider it instead of only those who have very few other options.