Novartis Files for Approval of Kymriah for Two Blood Cancers in the EU

Novartis Files for Approval of Kymriah for Two Blood Cancers in the EU

Novartis continues the process of making Kymriah (tisagenlecleucel) commercially available outside the U.S., now by applying with the European Medicines Agency (EMA) to approve the treatment for two types of cancer.

The submitted Marketing Authorization Application (MAA) covers the use of the CAR T-cell therapy for children and adolescents with B-cell acute lymphoblastic leukemia (ALL), and for adults with diffuse large B-cell lymphoma (DLBCL) who are not eligible for an autologous stem cell transplant. Both patient groups must have relapsed or refractory disease to be eligible for treatment.

“Since the historic FDA approval of Kymriah, formerly CTL019, we have launched, manufactured and supplied this highly individualized immunocellular therapy in a commercial setting and the submission to the EMA is a major step toward our goal of delivering it to more critically ill cancer patients around the world,” Vas Narasimhan, MD, global head of Drug Development and chief medical officer at Novartis, said in a press release.

“We look forward to working with the EMA to make CTL019 available to the children and adults who may benefit from this novel therapy,” added Narasimhan.

The application is based on data from the Phase 2 ELIANA trial (NCT02435849) in ALL, and the Phase 2 JULIET clinical trial (NCT02445248) in DLBCL.

The application also holds six-month results from the JULIET trial that Novartis will present at the American Society of Hematology (ASH) Annual Meeting in Atlanta in December.

Patients with these cancer types that have relapsed or are refractory to treatment face particularly poor outcomes. Untreated patients with relapsed or refractory DLBCL live for only three to four months, and only about 10 percent of children with relapsed or refractory ALL survive for five years, Novartis said.

Kymriah was approved in the U.S. for pediatric ALL in late August, and the company applied to expand the approval to include difficult-to-treat adult DLBCL in November.

“When tisagenlecleucel became a reality for certain patients and their families in the U.S. after approval by the FDA for patients with relapsed or refractory ALL this year, I believe it forever changed the face of cancer treatment,” said Stephen J. Schuster, MD, principal investigator of the JULIET trial.

“The data show this is a groundbreaking immunocellular therapy that has the potential to alter outcomes in patients who have limited options. This submission brings us closer to realizing that potential for more patients with fatal blood cancers,” he said.

Schuster also is the Robert and Margarita Louis-Dreyfus Professor in Chronic Lymphocytic Leukemia and Lymphoma Clinical Care and Research at University of Pennsylvania’s (UPenn) Perelman School of Medicine and director of the Lymphoma Program at the Abramson Cancer Center.

Novartis has developed Kymriah, which is a CAR T-cell therapy, in collaboration with researchers at UPenn. The JULIET and ELIANA trials also are performed in collaboration with the university.

Kymriah makes use of a patient’s own T-cells, which are gathered and genetically modified before being injected back into the patient.

The ELIANA trial showed that 83 percent of treated children had their cancer wiped out within three months of Kymriah treatment — an impressive result given the poor survival rates in this group. Meanwhile, 37 percent of DLBCL patients in the JULIET trials saw their cancer disappear.

“For patients in the EU living with these aggressive forms of blood cancer, we have very limited options to improve their chances of sustained remission after their disease has relapsed or become refractory to initial treatment,” said professor Gilles Salles, MD, PhD, Head of Hematology Department, Hospices Civils de Lyon, Lyon, France.

“The data for tisagenlecleucel has provided an optimistic look at the potential to achieve durable responses in two distinct and difficult-to-treat patient populations, helping to address a dire unmet need for patients.”

Novartis plans to submit additional applications for regulatory approval outside the U.S. and EU in 2018.

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