Advisory Committee Recommends That FDA Approve CTL019 for Treating Children with Leukemia

Advisory Committee Recommends That FDA Approve CTL019 for Treating Children with Leukemia

A U.S. Food and Drug Administration advisory committee has recommended that the agency approve Novartis’ CAR T-cell therapy CTL019 (tisagenlecleucel) as a treatment for children and young adults with leukemia.

The Oncologic Drugs Advisory Committee recommendation applies to patients with relapsed or refractory B-cell acute lymphoblastic leukemia, or ALL.

Panel members’ recommendation was unanimous — 10 to 0. Although the FDA can reject the recommendation, it will carry considerable weight when the agency decides whether to grant Novartis a Biologics License Application to market CTL019.

The agency has already granted priority review to the application. If approved, CTL019 will be the first CAR T-cell therapy it has authorized.

“The panel’s unanimous recommendation in favor of CTL019 moves us closer to potentially delivering the first-ever commercially approved CAR-T cell therapy to patients in need,” Bruno Strigini, the CEO of  Novartis Oncology, said in a press release.

“We’re very proud to be expanding new frontiers in cancer treatment by advancing immunocellular therapy for children and young adults with r/r [relapsed or refractory] B-cell ALL and other critically ill patients who have limited options. We look forward to working with the FDA as they complete their review.”

In weighing its recommendation, the advisory committee considered the results of clinical trials of CTL109, including the Phase 3 ELIANA study (NCT02435849). It was the first trial of a CAR T-cell therapy for children and adolescents whose objective was to obtain treatment approval.

Findings from another U.S. multicenter trial and a single-center trial supported the panel’s decision.

Novartis has developed CTL019 in collaboration with the University of Pennsylvania researchers who created it. Children’s Hospital of Philadelphia was the first institution to test the treatment in children.

“It is encouraging to see the FDA panel’s recommendation and continued momentum behind this innovative therapy, which has potential to help young patients with relapsed/refractory B-cell ALL,” said Dr. Carl June, who leads the University of Pennsylvania research team.

“We look forward to continuing to work with Novartis to help make a lasting impact on the way this disease is treated,” added June, who is director of two prestigious university research operations: the Center for Cellular Immunotherapies at the Perelman School of Medicine and the Parker Institute for Cancer Immunotherapy.

His colleague, Dr. Stephan Grupp, said there is a desperate need for innovative approaches to treating young people with the aggressive cancer. He has experienced that need first-hand through his work at the university and the children’s hospital.

“Today’s vote in favor of CTL019 is a positive step and we appreciate Novartis’ commitment to pediatric patients,” said Grupp, a pediatric professor at the Perelman School of Medicine and director of the children’s hospital’s Cancer Immunotherapy Frontier Program.

Novartis is also exploring CTL019 as a treatment for patients with relapsed or refractory diffuse B-cell lymphoma (DLBCL). The therapy received FDA Breakthrough Therapy Designation for that cancer in April 2017. The company will file approval-request documents for CTL109 as a DLBCL treatment this year.

The company is also conducting studies of CTL019 for a number of other forms of cancer.

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