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New Drug Approvals in Leukemia and Lymphoma Presented at ASH 2017

Laura Joszt
The FDA was busy in 2017, with a number of notable approvals, including the first chimeric antigen receptor T-cell treatment. In a session at the 59th American Society of Hematology Annual Meeting and Exposition in Atlanta, Georgia, employees from the FDA presented data on 5 new drug approvals in leukemia and lymphoma in 2017.
The FDA was busy in 2017, with a number of notable approvals, including the first chimeric antigen receptor (CAR) T-cell treatment. In a session at the 59th American Society of Hematology Annual Meeting and Exposition in Atlanta, Georgia, employees from the FDA presented data on 5 new drug approvals in leukemia and lymphoma in 2017.

Leukemia
Tisagenlecleucel was the first CAR T therapy to be approved and its approval was for the treatment of patients age 25 or younger who have B-cell precursor acute lymphoblastic leukemia (ALL). The approval was based on a single-arm international trial in patients with CD19 B-cell ALL. More than half (59%) of the patients had prior ALL hematopoietic stem cell transplantation (HSCT). The population in the study was a highly refractory and treatment-resistant population, explained Emily Jen, MD, PhD, of FDA.

While 78 patients were enrolled in the study, not all eligible patients received treatment due to manufacturing failures, patients dying before infusion, and some patients having an adverse event that precluded infusion. Of those who did receive the infusion, 63% achieved a complete response (CR) and the median duration of response had not been reached.

The treatment’s efficacy showed a substantial improvement over available therapy, such as blinatumumab, said Jen.

“However, treatment with tisagenlecleucel is not without some major safety concerns,” she said. The box includes warnings for cytokine release syndrome (CRS) and neurologic toxicities.

Treatment with tisagenlecleucel will also not be widely available. It will only be available through a restricted program with a Risk Evaluation and Mitigation Strategy (REMS). Facilities must be specially certified, providers must be trained to manage the risk, and patients must be educated.

The second leukemia drug presented was inotuzumab ozogamicin, a CD22-directed antibody-drug conjugate. It was approved for adult patients with CD22 relapsed or refractory ALL based on a trial with 326 patients enrolled. The study found CR in 35.8% of patients, with a median response of 8 months. Tanya Wroblewski, MD, of FDA, noted that it was interesting overall survival did not reach statistical significance.

She added that hepatic veno-occlusive disease (VOD) developed in 14% of patients, overall, and in 23% of those who underwent subsequent HSCT.

“The rationale behind the decision for the approval of inotuzumab considered the population of patients—adults with relapsed or refractory ALL—as having a high unmet medical need,” Wroblewski said. “Secondly, the durable complete remission can be considered evidence of the direct clinical benefit in patients with relapsed or refractory acute leukemias.”

The presentations by the FDA reviewers was followed by an overview from Crystal L. Mackall, MD, professor of pediatrics and internal medicine at Stanford University. There has been substantial progress in ALL, but it still represents a common disease with a high morbidity and mortality in patients and for patients, the outcomes remain poor, explained Mackall.

While the results of the inotuzumab trial were impressive, the development of liver toxicity offset the promise of those results a little. This is not isolated to inotuzumab. A previous agent, gemtuzumab ozogamicin, also demonstrated similar activity in ALL but the risk of hepatic VOD diminished the benefits of the agent.

In pediatric ALL, the efforts of the medical community have led to steadily improved outcomes. Tisagenlecleucel exemplifies the promise of new agents, Mackall said. The story of Emily Whitehead, who received 1 infusion of cells in 2012 that continue to circulate in her show the novelty and promise of the CAR T therapy.

“I want to argue that these CARs, despite the complexities in production and the cost, and these issues, are still poised for major impact in B-cell ALL,” she said.



 
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