The company did say that it's made an agreement with the US government to pay for the drug only when pediatric or young adult patients with the cancer respond to treatment by the end of their first month.
A multicenter trial of 63 pediatric and young adult patients with relapsed or refractory B-cell precursor ALL demonstrated the safety and efficacy of Kymriah.More news: Nigeria vs Cameroon: Rohr happy to have "full house"
In its announcement, Novartis said it plans to make additional filings for Kymriah in the USA and EU this year, including applications with the FDA and European Medicines Agency for the treatment of adult patients with r/r diffuse large B-cell lymphoma.
The disease is the most common type of childhood cancer in the US, making up 25 percent of cancer diagnoses for children under 15 years old, according the National Cancer Institute. "Not only does Kymriah provide these patients with a new treatment option where very limited options existed, but a treatment option that has shown promising remission and survival rates in clinical trials". Kymriah handles this turbocharging within the patients body. Once modified, the cells are infused back into the patient to kill the cancer cells. In the pivotal Novartis trial, 83 percent of patients achieved remission with a disease that has historically poor outcomes.
In the treatment, doctors collect the patient's own T-cells - one of the immune system's main cell types - and genetically reprograms them to target and attack leukemia cells. Tisagenlecleucel carries a boxed warning for cytokine release syndrome (CRS) and neurological events, which can be life threatening. However, there were some severe side effects observed in study. Other possible severe side effects could include serious infection, low blood pressure, kidney injury, fever and decreased oxygen. Kymriah's maker, Novartis, hasn't provided a price for the drug.More news: Fitbit expands product portfolio, launches a smartwatch, Bluetooth headphone and smart scale
At the time, American Cancer Society Deputy Chief Medical Officer Dr. Len Lichtenfeld said the real promise of these gene therapies is the potential to completely eradicate specific cancers both for the single patient as well as all their descendants.
Manufacturing and delivery are more complex in CAR-T therapies than for a typical drug.
The others still are being tracked to see how they fare long-term.More news: Hundreds of thousands march in Barcelona to show unity after Islamist attacks