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Recent FDA Approvals

February 2015 Vol 8, Special Issue: Payers' Perspectives in Oncology - FDA Approvals

Ibrutinib Gets New Indication for Waldenström’s Macroglobulinemia

On January 29, 2015, the US Food and Drug Administration (FDA) ap­­proved a new indication for ibrutinib (Imbruvica; Pharmacyclics) under its priority review process for the treatment of patients with Waldenström’s macroglobulinemia (WM), a rare type of non-Hodgkin lymphoma. Ibrutinib had previously received a breakthrough therapy designation by the FDA for this use.

WM progresses gradually over time, resulting from the overproduction of B-cells within the bone marrow, lymph nodes, liver, and spleen. These B-cells also overproduce immunoglobulin M, which may lead to bleeding and vision and nervous system problems. Ibrutinib inhibits the abnormal activity of these B-cells to fight WM.

The approval was based on a clinical study of 63 previously treated patients with WM. The patients received 420 mg of oral ibrutinib daily until disease progression or until side effects became intolerable. The overall response rate to the drug was 62%, with a response duration ranging from 2.8 months to approximately 18.8 months.

The most common adverse events with ibrutinib are thrombocytopenia, neutropenia, diarrhea, anemia, fatigue, musculoskeletal pain, bruising, nausea, upper respiratory tract infection, and rash.

Ibrutinib was initially approved by the FDA in November 2013 for the treatment of patients with mantle-cell lymphoma; in February 2014, it was approved for patients with previously treated chronic lymphocytic leukemia (CLL), and in July 2014, for patients with CLL and a deletion in chromosome 17.


Ruxolitinib Gets New Indication for Polycythemia Vera

On December 14, 2014, the FDA approved ruxolitinib (Jakafi; Incyte) for the treatment of patients with polycythemia vera (PV) who have had an inadequate response to or are intolerant of hydroxyurea. The approval was based on a randomized, multicenter, open-label, active-control trial of 222 patients with PV.

The composite end point was durable hematocrit control and spleen volume reduction, and a durable hematocrit control that obviated the need for regular phlebotomy.

Ruxolitinib was significantly better than best-available therapy in durable hematocrit control and in spleen volume reduction by week 32 (21% vs 1%; P <.001) and week 48 (19% vs 1%; ­­­P <.001), and had a 55% rate of durable hematocrit control by week 48.

The most common (incidence >20%) hematologic adverse reactions through week 32 were thrombocytopenia and anemia. The most common (incidence >10%) nonhematologic adverse events were headache, abdominal pain, diarrhea, dizziness, fatigue, pruritus, dyspnea, and muscle spasms. Overall, 4% of patients discontinued therapy because of adverse events.

The recommended starting dose of ruxolitinib is 10 mg twice daily, with modifications in some patient populations.


Blinatumomab First Immunotherapy Approved by the FDA for B-Cell ALL

On December 3, 2014, the FDA approved blinatumomab (Blincyto; Amgen) for the treatment of patients with relapsed or refractory Philadelphia chromosome (Ph)-negative precursor B-cell acute lymphoblastic leukemia (ALL), a rare and rapidly growing form of ALL. Blinatumomab is intended for use in patients with B-cell ALL. Blinatumomab is the first immunotherapy approved by the FDA for patients with leukemia. The drug acts as a connector between the CD19 protein (which is found on the surface of most B-cell lymphoblasts) and the CD3 protein found on T-cell lymphocytes, using the body’s T-cells to destroy the leukemia cells.

The FDA had initially granted this drug a breakthrough therapy designation, and applied its priority review and accelerated programs for this approval. The FDA is now requiring the manufacturer to conduct a new clinical trial to show a survival benefit with blinatumomab in patients with relapsed or refractory Ph-negative precursor B-cell ALL.

The approval was based on a clinical trial of 185 adults with Ph-negative relapsed or refractory precursor B-cell ALL. All patients received an infusion of blinatumomab for at least 4 weeks. Overall, 32% of patients had complete remission lasting approximately 6.7 months. The trial was not designed to show improvement in survival.

Blinatumomab is associated with significant risks, including cytokine release syndrome, encephalopathy, and nervous system events. It was approved with a Risk Evaluation and Mitigation Strategy program and has a boxed warning regarding these risks. The most common side effects reported were pyrexia, headache, peripheral edema, febrile neutropenia, nausea, hypokalemia, fatigue, constipation, diarrhea, and tremor.

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Last modified: August 30, 2021