Regulators on both sides of the Atlantic are undertaking speedy reviews of Novartis’ CAR-T therapy Kymriah (tisagenlecleucel; formerly CTL019).
In the US, the Food and Drug Administration has granted the therapy a priority review for use of the therapy to treat adults who are ineligible for or relapse after autologous stem cell transplant (ASCT).
In the EU, the European Medicines Agency has granted an accelerated assessment to Novartis’ application for approval of the therapy to treat children and young adults with r/r B-cell acute lymphoblastic leukaemia and adults with r/r DLBCL who are ineligible for ASCT.
If approved by the FDA and EMA, Kymriah would represent the first CAR-T therapy available for two distinct indications in non-Hodgkin lymphoma and B-cell ALL.
CAR-T offers a new treatment approach in that it is specifically manufactured for each individual patient. During the process, T cells are drawn from a patient's blood and reprogrammed in the lab to create T cells that are genetically coded to hunt the patient's cancer cells.
Kymriah became the first CAR-T cell therapy to receive regulatory approval when it was approved by the FDA in August last year for the treatment of patients up to 25 years of age with B-cell precursor ALL that is refractory or in second or later relapse.
Novartis says its first-in-class therapy showed an 83 percent overall remission rate in this patient population with limited treatment options and historically poor outcomes.
Hailing a new era in cancer treatment, Samit Hirawat, head of Novartis Oncology Global Drug Development, said the firm is now focused on working with the EMA and FDA “to bring this potentially transformative therapy to more patients."
Novartis says it also plans additional regulatory submissions for Kymriah in paediatric and young adult patients with r/r B-cell ALL and adult patients with r/r DLBCL beyond the US and EU this year.
The Swiss drug giant picked up rights to Kymriah under an agreement with the University of Pennsylvania in 2012, which also gives it worldwide rights to CAR-Ts developed in all cancer indications.
UK drugmaker Oxford BioMedica is the sole manufacturer of the lentiviral vector that encodes Kymriah.