On Friday, the U.S. Food and Drug Administration granted accelerated approval to Roche Holding’s Alecensa drug to treat people with a specific gene mutation.
The new drug will treat people with anaplastic lymphoma kinase (ALK)-positive metastatic, non-small cell lung cancer (NSCLC), whose disease has worsened after, or who could not tolerate treatment with Xalkori by Pfizer.
FDA approved Alecensa using the accelerated approval regulatory pathway, which allows the agency to approve products for serious or life-threatening diseases. The accelerated approval requirements requires a confirmatory study to verify and describe the clinical benefit of the drug.
Lung cancer is the leading cause of cancer death in the United States, with an estimated 221,200 new diagnoses and 158,040 deaths in 2015, shows the data from the National Cancer Institute.
California-based Genentech, a subsidiary of Roche Holding, said in a statement that an ALK gene mutation can occur in several different types of cancer cells, including lung cancer cells. ALK gene mutations are present in about 5% of patients with NSCLC, and in metastatic cancer, the disease spreads to new parts of the body. The brain is a common place for the disease to spread in ALK-positive NSCLC metastatic patients.
Alecensa is an oral medication that blocks the activity of the ALK protein, which may prevent NSCLC cells from growing and spreading.
According to a statement, experts studied safety and efficacy of Alecensa in two single-arm clinical trials of patients with metastatic ALK-positive NSCLC whose disease was no longer controlled by treatment with Xalkori. In the two trials, 61% of participants having measurable brain metastases experienced a complete or partial reduction in their brain tumors, lasting an average of 9.1 months.
There are some side effects of Alecensa, including fatigue, constipation, swelling (edema) and muscle pain (myalgia). The drug may cause serious side effects, including liver problems, severe or life-threatening inflammation of the lungs, very slow heartbeats and severe muscle problems. Moreover, treatment with Alecensa may cause sunburn when patients are exposed to sunlight.
“Today’s approval provides a new therapy for a group of patients who would have few treatment options once their disease no longer responds to treatment with Xalkori,” stated Richard Pazdur, director of the Office of Hematology and Oncology Products in the FDA’s Center for Drug Evaluation and Research. “In addition to the primary effect on tumors in the lung, Alecensa clinical trials provide evidence of an effect on tumors that had spread to the brain, which is an important effect for clinicians to understand.”