Leukemia drug gets US okay, struggles in UK

While FDA recently approved ibrutinib (Imbruvica, Janssen Biotech, Inc. and Pharmacyclics LLC) to treat patients with chronic lymphocytic leukemia (CLL), United Kingdom’s National Institute of Health and Care Excellence (NICE) will not yet recommend the medication.

Imbruvica is the first chemotherapy-free therapy alternative approved for treatment-naïve CLL patients by FDA. This newest indication also means that Imbruvica is now approved for use in all lines of CLL therapy, expanding the number of patients who may benefit from therapy.

The wholesale acquisition cost for 3 months of therapy with Imbruvica is $11,497.72.

At the same time, Janssen is "extremely disappointed" with draft guidance from NICE which did not recommend the drug as a treatment for CLL patients who aren't good candidates for chemo-immunotherapy. Currently available through the U.K.’s Cancer Drugs Fund (CDF), Imbruvica is the most requested CLL drug on the fund, demonstrating a "huge clinical demand,” Mark Hicken, Janssen U.K.'s managing director, said in a statement.

Related: FDA expands Opdivo use for metastatic melanoma

Imbruvica works as a potent and irreversible inhibitor of Bruton’s tyrosine kinase (BTK), which is an important signaling molecule in the B-cell receptor pathway. Activation of B-cell receptor signaling is important for the survival of malignant B-cells. By inhibiting BTK, Imbruvica prevents malignant B-cell proliferation and survival.

The newest FDA approval of Imbruvica was based on results from RESONATE-2, the first head-to-head trial comparing Imbruvica to a chemotherapy agent in the treatment of CLL. The study showed that Imbruvica extended progression-free survival (PFS) and overall response rate (ORR) in previously untreated patients with CLL aged 65 years or older when compared to chlorambucil.

Related: First chemotherapy antidote available soon

"People living with CLL who have not been previously treated now have an option that significantly improved progression-free survival when compared to the oral chemotherapy used in the RESONATE-2 trial," said Jan Burger, MD, PhD, associate professor in the Division of Cancer Medicine at the University of Texas MD Anderson Cancer Center, and RESONATE-2 study lead investigator. "The results seen in the RESONATE-2 clinical trial are truly compelling, and make this medicine an attractive first-line treatment option for clinicians in the hematology space."

Imbruvica is a capsule taken once daily with a glass of water. The most common adverse reactions associated with the use of Imbruvica in patients with B-cell malignancies include thrombocytopenia, diarrhea, anemia, neutropenia, and musculoskeletal pain. 

Related: FDA approves Gazyva sBLA with new data in previously untreated CLL