Early last week the Food and Drug Administration panel unanimously recommended the approval of the first CAR-T therapy, a pioneering new cancer drug from Novartis, marking a major medical milestone.
Novartis’ therapy is one of two cutting-edge treatments for blood cancers are poised to get approved by the end of the year.
The candidate, CTL019, is an investigational chimeric antigen receptor T cell (CAR-T) therapy, for the treatment of relapsed or refractory (r/r) pediatric and young adult patients with B-cell acute lymphoblastic leukemia (ALL).
Acute lymphoblastic leukemia comprises approximately 25% of cancer diagnoses among children under 15 years old and is the most common childhood cancer in the US. Effective treatment options for patients with r/r ALL are limited. In pediatric and young adult patients with B-cell ALL that have relapsed multiple times or become refractory to treatment, the five-year disease-free survival is less than 10-30%.
“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,” said Bruno Strigini, CEO, Novartis Oncology. “We’re very proud to be expanding new frontiers in cancer treatment by advancing immunocellular therapy for children and young adults with r/r 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.”The committee has asked Novartis to commit to 15 years of follow-up, given the therapy is following a fast-track approval and no long-term data is available yet. The short-term results, however, have convinced the experts that it’s worth giving CTL019 a try: after 12 months, 79%of patients with acute lymphoblastic leukemia (ALL) were still alive, and 75% had not relapsed again.
CAR-T therapy, which several other companies are also pursuing drugs for, works by extracting and isolating a patient’s T cells, genetically altering them, and then infusing them back into the patient’s body.
The following month, Kite Pharma, the other company in the race, is expected to get an answer from the FDA. That’s for Kite’s CAR-T treatment for aggressive B-cell non-Hodgkin lymphoma (more general than DLBCL).
Upon approval, Novartis has said it plans a slow roll-out because of serious side effects that result from the therapy, mainly making it available to only a few dozen medical establishments.
One of the big issues in CAR-T cell therapy — the cost, which analysts say could be in the hundreds of thousands of dollars — wasn’t discussed because that is beyond the FDA’s purview. Novartis hasn’t released pricing information.
