FDA Approves Alpelisib Combination Therapy for Advanced Breast Cancer
Posted: Tuesday, May 28, 2019
On May 24, 2019, the U.S. Food and Drug Administration (FDA) approved alpelisib (Piqray) tablets, to be used in combination with the FDA-approved endocrine therapy fulvestrant, to treat postmenopausal women and men with hormone receptor–positive, HER2-negative, PIK3CA-mutated, advanced or metastatic breast cancer, as detected by an FDA-approved test following disease progression on or after an endocrine-based regimen.
“[Alpelisib] is the first PI3K inhibitor to demonstrate a clinically meaningful benefit in treating patients with this type of breast cancer,” stated 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.
The FDA also approved the companion diagnostic test, therascreen PIK3CA RGQ PCR Kit, to detect the PIK3CA mutation in a tissue and/or a liquid biopsy. Patients who test negative by the therascreen test using the liquid biopsy should undergo tumor biopsy for PIK3CA mutation testing.
The efficacy of alpelisib was studied in the SOLAR-1 trial (ClinicalTrials.gov identifier NCT02437318), a randomized trial of 572 postmenopausal women and men with hormone receptor–positive, HER2-negative, advanced or metastatic breast cancer whose cancer had progressed while on or after receiving an aromatase inhibitor. The trial results showed the addition of alpelisib to fulvestrant significantly prolonged progression- free survival (median of 11 months vs. 5.7 months) in patients whose tumors had a PIK3CA mutation.
Common side effects of alpelisib are high blood sugar levels, increase in creatinine, diarrhea, rash, decrease in lymphocyte count in the blood, elevated liver enzymes, nausea, fatigue, low red blood cell count, increase in lipase, decreased appetite, stomatitis, vomiting, weight loss, low calcium levels, activated partial thromboplastin time, and hair loss.
