The U.S. Food and Drug Administration (FDA) has granted accelerated approval to Imbruvica (ibrutinib) for patients with marginal zone lymphoma (MZL) who have received at least one prior therapy containing anti-CD20 agents and who require systemic therapy.
“Patients with relapsed/refractory marginal zone lymphoma are in critical need of treatment options to manage living with this rare, serious blood cancer,” Ariela Noy, MD, hematologic oncologist at Memorial Sloan Kettering Cancer Center in New York and lead investigator of the Phase 2 study that led to the approval, said in a news release.
“This approval of Imbruvica represents a welcome new oral option for the MZL community and is the first approved therapy for these patients,” she said.
The FDA’s decision was based on results from the PCYC-1121 Phase 2 trial (NCT01980628), recently presented at the 58th Annual American Society of Hematology (ASH) Meeting in December.
The open-label, multicenter, single-arm study was designed to assess the safety and effectiveness of Imbruvica in patients with all three subtypes of MZL: mucosa-associated lymphoid tissue (MALT), nodal MZL (NMZL), and splenic MZL (SMZL).
After a median follow-up of 19.4 months, 3.2% of patients achieved a complete response and 42.9% achieved a partial response, accounting for an overall response rate of 46%.
All three MZL subtypes responded similarly to the therapy, with MALT, NMZL, and SMZL patients showing an overall response rate of 46.9%, 41.2%, and 50.0%, respectively.
Patients took a median of 4.5 months until initial response to treatment, and median duration of response was not reached.
Based on the promising overall response rates, which demonstrated that nearly half of patients respond to the therapy, the FDA granted accelerated approval to Imbruvica. However, continued approval of this therapy is dependent on continued verification of clinical benefit in a confirmatory trial for this patient subset, for which there are no other FDA-approved therapies.
“This approval broadens the indication for Imbruvica to include relapsed/refractory marginal zone lymphoma. It addresses a great unmet need for patients living with this serious blood cancer,” said Peter F. Lebowitz, MD, PhD, global oncology head of Janssen.
“Building on our longstanding commitment of providing meaningful treatment solutions for people living with hematological malignancies, this milestone marks the fifth blood cancer indication for this medication,” he said. “We continue to research its potential across a range of B-cell malignancies based on the drug’s mechanism of action.”