Novartis was granted Regenerative Medicine Advanced Therapy (RMAT) designation by the U.S. FDA for 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 pediatric and young adult acute lymphoblastic leukemia (ALL), and r/r adult diffuse large B-cell lymphoma (DLBCL).

The RMAT designation program is part of the 21st Century Cures Act. The program was created to expedite the development and review of regenerative medicine therapies intended to treat, modify, reverse or cure a serious condition. 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.

Follicular lymphoma, the second most common form of non-Hodgkins lymphoma (NHL) and represents approximately 22% of NHL cases. Despite new treatments that improve overall survival, FL is regarded as an incurable malignancy with a relapsing and remitting pattern. There also is an unmet need in people who are refractory to treatment or quickly relapse, who may exhaust treatment options while they are still healthy enough to receive active treatment.