A NOVEL TREATMENT FOR MANTLE CELL LYMPHOMA

The U.S. Food and Drug Administration (FDA) today granted accelerated approval to acalabrutinib (Calquence) for the treatment of adults with mantle cell lymphoma who have received at least one prior therapy. “Mantle cell lymphoma is a particularly aggressive cancer,” said Richard Pazdur, MD, Director of the FDA’s Oncology Center of Excellence and Acting Director of the Office of Hematology and Oncology Products in the FDA’s Center for Drug Evaluation and Research. “For patients who have not responded to treatment or have relapsed, [acalabrutinib] provides a new treatment option that has shown high rates of response for some patients in initial studies.”

Acalabrutinib is a kinase inhibitor that works by blocking an enzyme needed by the cancer to multiply and spread. The drug was approved using the Accelerated Approval pathway, under which the FDA may approve drugs for serious conditions where there is unmet medical need and a drug is shown to have certain effects that are reasonably likely to predict a clinical benefit to patients. Further study is required to verify and describe anticipated clinical benefits of acalabrutinib, and the sponsor is currently conducting this trial.

Pivotal Trial

Today’s approval of acalabrutinib was based on data from a single-arm trial that included 124 patients with mantle cell lymphoma who had received at least one prior treatment. The trial measured overall response rate, and 81% of patients enrolled had a complete or partial response (40% complete response, 41% partial response).

Common side effects of acalabrutinib include headache, diarrhea, bruising, fatigue, myalgia, anemia, thrombocytopenia, and neutropenia.

Serious side effects include hemorrhage, infections, and atrial fibrillation. Second primary malignancies have occurred in some patients taking acalabrutinib. Women should discontinue breastfeeding during acalabrutinib therapy.

The FDA granted this application Priority Review and Breakthrough Therapy designations. Acalabrutinib also received Orphan Drug designation, which provides incentives to assist and encourage the development of drugs for rare diseases.