In January 2018, the U.S. FDA (Food and Drug Administration) accepted Novartis’s (NVS) supplemental biologics license application (or SBLA) for Kymriah suspension for the treatment of adults with relapsed or refractory diffuse large B-cell lymphoma (or DLBCL) who are not candidates for autologous stem cell transplant (or ASCT). The U.S. FDA granted Novartis’s sBLA for priority review.
Also, the European Medicines Agency (or EMA) granted accelerated assessment to the marketing authorization application (or MAA) for Novartis’s Kymriah for the treatment of children and young adults with relapsed or refractory B-cell acute lymphoblastic leukemia (or ALL) and also for adults with relapsed or refractory DLBCL who are not candidates for ASCT.
If the FDA and EMA approve Kymriah, then it will be the first chimeric antigen receptor T-Cell (or CAR-T) therapy available for two different indications in non-Hodgkin lymphoma and B-cell ALL.
Clinical trials for Kymriah
Regulatory submissions to the FDA and the European Commission (or EC) were based on data from the global clinical trial program of Kymriah in children and young adults with relapsed or refractory B-cell ALL and adults with relapsed or refractory DLBCL. Novartis conducted the pivotal phase 2 Juliet trial, which evaluated the safety and efficacy of Kymriah for the treatment of adults with relapsed or refractory DLBCL. Novartis also conducted the pivotal phase 2 Eliana trial, which evaluated the safety and efficacy of Kymriah for the treatment of pediatric patients with relapsed or refractory B-cell ALL. The data from the phase 2 JULIET trial and phase 2 ELIANA trial served as the basis of the sBLA and MAA submissions to the U.S. FDA and EC, respectively.
FDA and EC approvals for label expansion are expected to further boost Kymriah’s sales growth. Presently, various chemotherapies and Roche’s Rituxan are used for the treatment of relapsed or refractory DLBCL. Some other notable drugs under investigation for the treatment of DLBCL include Novartis’s (NVS) Afinitor and Farydak, Seattle Genetics’ (SGEN) Adcetris, Bristol-Myers Squibb’s (BMY) Opdivo, and CTI Biopharma’s (CTIC) Pixuvri. The Vanguard Total International Stock ETF (VXUS) invests ~0.93% of its total portfolio holdings in Novartis.