The US Food and Drug Administration’s (FDA’s) announcement makes Kymriah (tisagenlecleucel) the only chimeric antigen receptor (CAR) T-cell therapy to have received US regulatory approval for two distinct indications.
Kymriah – made at Novartis’ Morris Plains, New Jersey, US facility – is a one-time treatment manufactured using the patient’s own T-cells, which have been genetically modified to target and kill leukaemia cells.
The $475k ($397k) treatment was the first CAR-T therapy to receive regulatory approval in August last year. At that time, Kymriah was solely indicated to treat certain paediatric and young adult patients with a form of acute lymphoblastic leukaemia (ALL).
The latest approval will see the autologous CAR T-cell therapy be intravenously infused to treat adult patients with relapsed or refractory (r/r) large B-cell lymphoma after two or more lines of systemic therapy.
“Novartis has set a price of $373,000 for this novel therapy and is confident in the value of Kymriah for patients with r/r DLBCL,” spokesperson Eric Althoff told us.
The large B-cell lymphoma Kymriah indication has been priced the same as Kite's Yescarta (axicabtagene ciloleucel), he noted.
Novartis looks to Europe and Japan
According to Althoff, Novartis is looking to market Kymriah in various geographical locations this year.
In November 2017, the firm submitted a marketing authorisation application (MAA) for Kymriah to the European Medicines Agency (EMA), for two indications.
“The application is for the treatment of children and young adults with relapsed or refractory (r/r) B-cell acute lymphoblastic leukemia (ALL) and for adult patients with r/r diffuse large B-cell lymphoma (DLBCL) who are ineligible for autologous stem cell transplant (ASCT),” Althoff told us.
“This application has been granted accelerated assessment and is currently under review,” he added.
Last month, Novartis filed a regulatory application in Japan for pediatric ALL and r/r DLBCL.
“Additional filings beyond the US and EU are anticipated this year,” we were told.