Regulatory Affairs

Novartis gets FDA nod for cancer treatment Kymriah

PBR Staff Writer Published 31 August 2017

Novartis has secured approval from the US Food and Drug Administration (FDA) for its Kymriah (tisagenlecleucel or CTL019) to treat children and young adults with B-cell precursor acute lymphoblastic leukemia (ALL).

Claimed to be the first of its kind approval, the FDA has approved Kymriah to treat patients up to 25 years of age with B-cell precursor ALL, which is refractory or in second or later relapse.

Kymriah is the chimeric antigen receptor T cell (CAR-T) therapy and one-time treatment that uses a patient's own T cells to treat cancer.

According to the company, Kymriah is the first therapy based on gene transfer approved by the FDA.

In 2012, Novartis collaborated with the University of Pennsylvania (Penn) to further research, develop and commercialize CAR-T cell therapies, including Kymriah, for the investigational treatment of cancers.

Novartis will use its New Jersey facility to manufacture Kymriah for each individual by using their own cells.

The approval was based on data from the pivotal open-label, multicenter and single-arm Phase II ELIANA trial.

ELIANA trial is said to be the first pediatric global CAR-T cell therapy registration trial, which evaluated patients in 25 centers in the US, EU, Canada, Australia and Japan.

In April, the company received FDA breakthrough therapy status for CTL019 based on data from the JULIET study, which is the multi-center global registration study for CTL019 in adult patients with r/r DLBCL.

Novartis CEO Joseph Jimenez said: “Five years ago, we began collaborating with the University of Pennsylvania and invested in further developing and bringing what we believed would be a paradigm-changing immunocellular therapy to cancer patients in dire need.

“With the approval of Kymriah, we are once again delivering on our commitment to change the course of cancer care."

Novartis Oncology CEO Bruno Strigini said: "We are so proud to be part of this historic moment in cancer treatment and are deeply grateful to our researchers, collaborators, and the patients and families who participated in the Kymriah clinical program.”

Image: Novartis headquarters in Basel, Switzerland. Photo: courtesy of Novartis AG.