The European Commission (EC) has approved Novartis’ Kymriah (tisagenlecleucel), a CAR-T cell therapy, for the treatment of adult patients with relapsed or refractory (r/r) follicular lymphoma (FL) after two or more lines of systemic therapy.
The approval follows a positive opinion in March by the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) and is applicable to all 27 European Union member states plus Iceland, Norway and Liechtenstein. This approval marks the third indication for Kymriah and makes it the first CAR-T cell therapy approved in the EU for these patients, which include those with r/r FL grade 1, 2 and 3A.
“When follicular lymphoma fails to respond to treatment or comes back, it is typically more aggressive and difficult to treat; patients often end up cycling through multiple lines of therapy with decreasing benefit,” said Catherine Thieblemont, MD, PhD, Professor of Hematology in the Paris VII- University, France and Head of the Hemato-Oncology Unit of St-Louis Hospital in Paris. “The approval of Kymriah in Europe brings patients closer to a potentially definitive therapy, providing us hope for improved outcomes.”
The approval is based on the global Phase II ELARA trial showing that 86% of patients who were treated with Kymriah had a response, including 69% who had a complete response (CR). Prolonged durable response to treatment was demonstrated with an estimated 87% of patients who achieved a CR still in response at or more than nine months after initial response. In the trial, 94 infused patients were evaluated for efficacy with a median follow-up of approximately 21 months.
Among 97 patients evaluable for safety, the safety profile of Kymriah was remarkable. Cytokine release syndrome (CRS) was reported in 50% of patients after Kymriah infusion, and no Grade 3 or 4 events were reported, as defined by the Lee scale. Neurological adverse reactions occurred in 9% of patients (1% were Grade 3 or 4) within eight weeks after Kymriah infusion. Severe infections (Grade 3 or 4) occurred in 16% of patients.
“With this approval, we are pleased to be able to offer this transformative therapy to more people across the globe living with this advanced blood cancer,” said Marie-France Tschudin, President, Innovative Medicines International & Chief Commercial Officer, Novartis. “With long-lasting responses and a safety profile that allows for flexible administration, we are striving to rewrite cancer survival and alleviate the burden of this disease for patients and the healthcare system.”
In addition to r/r FL, Kymriah is approved for the treatment of pediatric and young adult patients up to and including 25 years of age with B cell acute lymphoblastic leukemia (ALL) that is refractory, in relapse post-transplant or in second or later relapse, and adult patients with r/r diffuse large B cell lymphoma (DLBCL) after two or more lines of systemic therapy.