FDA approves single-agent treatment for chronic lymphocytic leukemia

February 12, 2014

FDA expanded the approved use of ibrutinib (Imbruvica, Pharmacyclics and Janssen Biotech) as a single agent, oral kinase inhibitor for chronic lymphocytic leukemia (CLL) patients who have received at least one previous therapy.

FDA expanded the approved use of ibrutinib (Imbruvica, Pharmacyclics and Janssen Biotech) as a single agent, oral kinase inhibitor for chronic lymphocytic leukemia (CLL) patients who have received at least one previous therapy.

CLL is a type of cancer that starts from white blood cells (lymphocytes) in the bone marrow. CLL mainly affects older adults, and accounts for about one-third of all leukemias. According to the American Cancer Society, estimates for leukemia in the United States for 2014 are:

About 52,380 new cases of leukemia and about 24,090 deaths from leukemia (all kinds)

About 15,720 new cases of chronic lymphocytic leukemia (CLL)

About 4,600 deaths from CLL

Ibrutinib works by blocking the enzyme that allows cancer cells to grow and divide. In November 2013, FDA granted ibrutinib accelerated approval to treat patients with mantle cell lymphoma, a rare and aggressive type of blood cancer, if those patients received at least one prior therapy.

Ibrutinib for CLL also received priority review and orphan-product designation because the drug demonstrated the potential to offer significant improvement in safety or effectiveness in the treatment of a serious condition and is intended to treat a rare disease, respectively.

FDA’s accelerated approval of  ibrutinib for CLL is based on a clinical study of 48 previously treated participants. On average, participants were diagnosed with CLL 6.7 years prior to the study and had received four previous therapies. All study participants received a 420-mg orally administered dose of ibrutinib until the treatment reached unacceptable toxicity or the disease progressed. Nearly 58% of participants had their cancer shrink after treatment (overall response rate), results showed. At the time of the study, the duration of response ranged from 5.6 to 24.2 months. An improvement in survival or disease-related symptoms has not been established.

In the clinical study, the most common side effects observed include thrombocytopenia, diarrhea, bruising, neutropenia, anemia, upper respiratory tract infection, fatigue, musculoskeletal pain, rash, pyrexia, constipation, peripheral edema, arthralgia, nausea, stomatitis, sinusitis, and dizziness.

“The clinical data on Imbruvica for the treatment of chronic lymphocytic leukemia includes only ‘surrogate’ end points-in this case, cancer shrinkage versus increases in survival or life years,” said John E. Schneider, PhD, CEO, Avalon Health Economics LLC. “The drug will still have to prove itself in terms of survival, but FDA, for the time being, is of the opinion that the cancer shrinkage data is sufficient to warrant getting this to patients who need it.”

“Relapsed or refractory chronic lymphocytic leukemia can be challenging to treat and therapy options currently available are not ideal for all patients. Imbruvica, a once-daily oral medication, represents a major achievement in drug development and an important option for patients with this disease,” according to Gary Rice, RPh, Vice President of Clinical Services at Diplomat, an independent specialty pharmacy, and one of the distributors of the medication for the treatment of mantle cell lymphoma.