Novartis' Kymriah posts strong 5-year data in ALL after setback in label expansion bid

Five years ago, Novartis’ Kymriah made history when it became the first gene therapy approved for use in the United States.

There have been some ups and downs for the treatment as Novartis has tried to expand its use in the lymphoma arena. On Monday, the company unveiled an up—positive data from a long-term, follow-up study of its use in its first approved indication.

Of 79 children and young adult patients with relapsed or refractory B-cell acute lymphoblastic leukemia (ALL), 55% of them survived during the five-year study. For patients in remission, the five-year relapse-free survival (RFS) rate was 44% and the median RFS was 43 months.

The median event-free survival for patients in remission within three months of infusion was 43.8 months. Events here are defined as no response, fast relapse or relapse from complete remission.

The findings, which were presented during the European Hematology Association Hybrid Congress, show the curative potential of Kymriah in a disease with few treatment options, Novartis said.

Before the approval of Kymriah, patients had a five-year survival rate of less than 10%, Stephen Grupp, M.D., Ph.D., Director of the Susan S. and Stephen P. Kelly Center for Cancer Immunotherapy at Children’s Hospital of Philadelphia, said in a statement. He called Kymriah a “game-changing option.”

“These data mark a moment of profound hope,” Grupp added, “as relapse after five years is rare.”   

Another finding in the study was that 82% of patients showed complete remission or complete remission with incomplete hematologic recovery within three months of infusion. Additionally, for patients in remission, the five-year relapse-free survival rate was 49%.

Last month, Kymriah won FDA approval for relapsed or refractory follicular lymphoma. In 2018, it also was endorsed by the U.S. regulator for some large B-cell lymphoma patients.

The treatment was expected to perform well in earlier-stage lymphoma but last year it showed no benefit versus standard of care in aggressive B-Cell non-Hodgkin lymphoma.

Meanwhile, Gilead’s Yescarta scored an approval in the indication in April of this year, 14 months after Bristol Myers Squibb’s Breyanzi did the same.

EDITORS NOTE: Story was updated with corrected trial data from the company.