publication date: Jul. 14, 2017
ODAC unanimously recommends approval for CAR T-cell therapy for relapsed and refractory B-cell ALL in kids and young adults
By Claire Dietz and Paul Goldberg
“If you want to see what a cure looks like, you already have,” said Tom Whitehead as his daughter Emily joined him at the lectern at the public hearing of the FDA Oncologic Drugs Advisory Committee July 12.
“She’s standing right beside me.”
Moments later, after the agency’s clinical advisors voted unanimously 10-0 to recommend approval for CTL019 (tisagenlecleucel), the Novartis agent was set on its way to become the first Chimeric Antigen Receptor T-cell therapy to be approved by FDA.
CTL019, dubbed “a living drug,” is likely to be indicated for pediatric and young adult patients 3 to 25 years of age with relapsed/refractory B-cell acute lymphoblastic leukemia.
On April 17, 2012, at Children’s Hospital of Philadelphia, Emily, then 7, became Patient One to receive CTL019.
With two recurrences, Emily was out of treatment options and home hospice was the only thing left. Under normal circumstances, it’s wrong to expect a dramatic benefit in a phase I study, but within 23 days of starting CTL019, Emily’s disease disappeared.
In the pivotal phase II study presented to ODAC, the overall remission rate was reported at 83 percent, with 75 percent of patients remaining relapse-free at six months after the remission onset. The survival probability was 89 percent at six months and 79 percent at 12 months. The median overall survival was about 16.6 months.
Slam dunk applications usually get approved without ODAC. However, when new mechanisms of action are involved, as is the case here,the agency usually consults its advisors. This was the first application to be reviewed jointly by the FDA Oncology Center of Excellence and the agency’s Center … Continue reading ODAC unanimously recommends approval for CAR T-cell therapy for relapsed and refractory B-cell ALL in kids and young adults
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