Rare Disease Report

FDA Approves Zykadia (ceritinib) As First Line Therapy for Rare Lung Cancer

MAY 26, 2017
James Radke
The US Food and Drug Administration (FDA) has approved the expanded use of Zykadia (ceritinib) from being second-line treatment of patients with metastatic non-small cell lung cancer (NSCLC) whose tumors are anaplastic lymphoma kinase (ALK)-positive to first-line treatment.
In September 2013, the FDA granted ceritinib with orphan drug designation for ALK-positive NSCLC and in April, 2014, the FDA approved the drug for treating ALK-positive NSCLC patients who had progressed on, or are intolerant to, crizotinib.  Approximately 3-7% of all patients with NSCLC have an ALK gene rearrangement.
The approval of ceritinib as first-line treatment is based on an open-label, randomized, multicenter, global, Phase 3 trial demonstrating that patients treated with ceritinib had a median progression-free survival (PFS) of 16.6 months (95% confidence interval [CI]: 12.6, 27.2), compared to 8.1 months (95% CI: 5.8, 11.1) for patients treated with standard first-line pemetrexed-platinum chemotherapy with pemetrexed maintenance.
The drug was even more effective in ALK-positive NSCLC without brain metastases. In the Phase 3 study, 121 of the 376 patients had brain metastases while 255 patients did not. And in the group without brain metastases, patients treated with first-line ceritinib had a median PFS of 26.3 months (95% CI: 15.4, 27.7), compared to 8.3 months (95% CI: 6.0, 13.7) for patients treated with chemotherapy (HR = 0.48 [95% CI: 0.33, 0.69]). Among patients with brain metastases, the ceritinib-treated group had a median PFS of 10.7 months (95% CI: 8.1, 16.4) compared to 6.7 months (95% CI: 4.1, 10.6) in the standard chemotherapy group (HR = 0.70 [95% CI: 0.44, 1.12]).
The most common adverse reactions in phase 3 trial were diarrhea (85%), nausea (69%), vomiting (67%), fatigue (45%), abdominal pain (40%), decreased appetite (34%) and cough (25%).   Most common Grade 3/4 adverse reactions were fatigue (7%), vomiting (5%), diarrhea (4.8%), abdominal pain (3.7%), weight loss (3.7%), nausea (2.6%) and prolonged QT interval (2.6%).

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