The FDA has granted Regenerative Medicine Advanced Therapy (RMAT) designation to Novartis’ Kymriah (tisagenlecleucel) for an investigational new indication to treat patients with relapsed or refractory (r/r) follicular lymphoma (FL).
Kymriah, which is designed to be a one-time treatment, is the first-ever FDA-approved CAR-T cell therapy.
The potential approval in r/r FL will be the third indication for Kymriah, which also has indications in r/r paediatric and young adult acute lymphoblastic leukaemia (ALL), and r/r adult diffuse large B-cell lymphoma (DLBCL).
“This designation supports the advancement of Kymriah, which could potentially address an unmet need in certain patients with follicular lymphoma, as we strive to reimagine medicine at Novartis,” said John Tsai, Head of Global Drug Development and Chief Medical Officer of Novartis.
“These patients are often faced with the burden of several years of various treatments as their disease continues to progres.”
The FDA granted RMAT designation for Kymriah in FL based on preliminary clinical evidence from the ELARA clinical trial, an ongoing multi-center, phase II study to determine the efficacy and safety of tisagenlecleucel in adult patients with relapsed or refractory FL.
Kymriah was developed in collaboration with the Perelman School of Medicine at the University of Pennsylvania, a strategic alliance between industry and academia which was first-of-its-kind in CAR-T research and development.