FDA takes second action under international collaboration, approves new treatment option for patients with chronic lymphocytic leukemia

FDA News Release

• For Immediate Release: November 21, 2019

Today, as part of Project Orbis, a collaboration with the Australian Therapeutic Goods Administration (TGA) and Health Canada, the U.S. Food and Drug Administration granted supplemental approval to Calquence (acalabrutinib) for the treatment of adults with chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL). This new approved indication for Calquence provides a new treatment option for patients with CLL or SLL as an initial or subsequent therapy.

“Today, as part of a U.S., Australian and Canadian collaboration known as Project Orbis, the U.S. approved a new treatment option for those living with chronic lymphocytic leukemia or small lymphocytic lymphoma. The FDA’s Project Orbis provides a framework for concurrent submission and review of oncology drug applications among the FDA’s international partners,” said Richard Pazdur, M.D., director of the FDA’s Oncology Center of Excellence and acting director of the Office of Oncologic Diseases in the FDA’s Center for Drug Evaluation and Research. “We are pleased to continue working alongside our Australian and Canadian colleagues to facilitate new treatment options for patients and the FDA looks forward to working with other countries in future application reviews.”

CLL and SLL are similar cancers, but they occur in different areas of the body. CLL occurs mainly in the blood and bone marrow, while SLL occurs mainly in the lymph nodes. Both are cancers of lymphocytes, which are a type of immune cell that helps the body fight infection. Symptoms of CLL or SLL include low red blood cell counts (anemia), low platelet counts, fatigue, enlarged lymph nodes, and a higher risk of infection.

The supplemental approval of Calquence for patients with CLL or SLL was based on two randomized clinical trials that compared Calquence to other standard treatments. The first clinical trial involved 535 patients with previously untreated CLL. Patients receiving Calquence had a longer progression-free survival (the amount of time a patient stays alive without the cancer growing) compared to patients receiving other standard treatments. The second clinical trial included 310 patients with previously treated CLL. Patients receiving Calquence also had a longer progression-free survival than patients receiving other standard treatments.

The most common side effects of Calquence were anemia, neutropenia (abnormally low levels of white blood cells), upper respiratory tract infection, thrombocytopenia (abnormally low levels of platelets), headache, diarrhea and musculoskeletal pain (pain that affects the muscles and bones). Patients may experience atrial fibrillation and flutter and should be monitored for symptoms of arrhythmias (irregular heart beat). Patients may experience serious infections and should be monitored and treated promptly. Patients should also be monitored for bleeding and managed appropriately. Patients may also experience low blood counts and should have blood work monitored regularly. Patients should be advised to use sun protection as other malignancies, such as skin cancers and other solid tumors, have occurred in patients taking Calquence.

The FDA advises health care professionals to tell females of reproductive age to use effective contraception during treatment with Calquence. Women who are pregnant or breastfeeding should not take Calquence because it may cause harm to a developing fetus or newborn baby, or cause delivery complications.

In addition to the international collaboration with Australia and Canada, this review used the Real-Time Oncology Review (RTOR) pilot program, which can streamline the submission of data prior to the completion and submission of the entire clinical application. RTOR, and its accompanying Assessment Aid, facilitated discussions among the regulatory agencies. These applications were approved four months prior to the FDA goal date. The FDA granted this application Priority Review and Breakthrough Therapy designation. The FDA granted approval of Calquence to AstraZeneca.

The FDA, an agency within the U.S. Department of Health and Human Services, protects the public health by assuring the safety, effectiveness, and security of human and veterinary drugs, vaccines and other biological products for human use, and medical devices. The agency also is responsible for the safety and security of our nation’s food supply, cosmetics, dietary supplements, products that give off electronic radiation, and for regulating tobacco products.