Novartis announced that the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) have accepted the company’s Supplemental Biologics License Application (sBLA) and Type II Variation, respectively, for Kymriah (tisagenlecleucel) in adult patients with relapsed or refractory (r/r) follicular lymphoma (FL) after two prior lines of treatment. The FDA has also granted priority review to the company’s sBLA for Kymriah in adult patients with r/r FL. Kymriah was previously granted orphan medicinal product designation by the European Commission (EC) for FL. If approved in this potential third indication, Kymriah would have the opportunity to present an important treatment option for those patients with r/r FL in need of potentially definitive outcomes.

The regulatory submissions are based on positive data from the pivotal Phase II ELARA trial, which investigated the efficacy and safety of Kymriah in adult patients with r/r FL. The trial met the primary endpoint with robust responses observed in heavily pretreated patients. The safety profile was remarkable, with no patients experiencing grade 3 or higher cytokine release syndrome (CRS) related to Kymriah within the first 8 weeks following infusion¹. Data from the trial was presented earlier this year as an oral presentation during the 2021 Annual American Society of Clinical Oncology (ASCO) Virtual Scientific Meeting.

“This is an important milestone in our mission to bring Kymriah to adult patients with relapsed or refractory follicular lymphoma. Receiving orphan drug designation from the EC as well as priority review from the FDA underscores the unmet need and urgency for these patients. With Kymriah demonstrating impressive results in the ELARA trial, we are hopeful that we can offer a unique and potentially definitive treatment that minimizes the burden,” said Jeff Legos, Executive Vice President, Global Head of Oncology & Hematology Development, Novartis.

Kymriah is currently approved by the FDA, EMA and other regulatory authorities for the treatment of r/r pediatric and young adult (up to and including 25 years of age) acute lymphoblastic leukemia (ALL), and r/r adult diffuse large B-cell lymphoma (DLBCL).