Kymriah receives RMAT designation for follicular lymphoma
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The FDA granted regenerative medicine advanced therapy designation to tisagenlecleucel — a chimeric antigen receptor T-cell therapy — for patients with relapsed or refractory follicular lymphoma, according to a press release issued by the manufacturer.
Tisagenlecleucel (Kymriah, Novartis) is a genetically modified anti-CD19 autologous CAR T-cell therapy.
The FDA designation is based on preliminary data from the ELARA trial, an ongoing multicenter, phase 2 study to determine the efficacy and safety of tisagenlecleucel for adults with relapsed or refractory follicular lymphoma.
“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,” John Tsai, MD, head of global drug development and chief medical officer at Novartis, said in the release. “These patients are often faced with the burden of several years of various treatments as their disease continues to progress.”
The FDA’s regenerative medicine advanced therapy designation program is part of the 21st Century Cures Act. The program aims to expedite review of regenerative medicine therapies intended to treat, modify, reverse or cure serious or life-threatening diseases or conditions. Eligible therapies must have preliminary clinical evidence of their potential to address unmet medical needs for such diseases or conditions.
Novartis plans to submit a U.S. regulatory filing for Kymriah in relapsed or refractory follicular lymphoma next year. If approved, it would be the third B-cell malignancy indication for tisagenlecleucel, which has been approved by the FDA for children and young adults with relapsed or refractory acute lymphoblastic leukemia and adults with relapsed or refractory diffuse large B-cell lymphoma.