New Therapy for Breast Cancer Granted FDA Approval

Officials with the FDA have approved alpelisib (Piqray, Novartis) tablets, to be used in combination with the endocrine therapy fulvestrant, to treat postmenopausal women, and men, with hormone receptor (HR)-positive, human epidermal growth factor receptor 2 (HER2)-negative, PIK3CA-mutated, advanced or metastatic breast cancer following progression on or after an endocrine-based regimen.

These tablets are the first PI3K inhibitor to demonstrate a clinically meaningful benefit in treating patients with this type of breast cancer, according to 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 ability to target treatment to a patient’s specific genetic mutation or biomarker is becoming increasingly common in cancer treatment, and companion diagnostic tests assist oncologists in selecting patients who may benefit from these targeted treatments,” Pazdur said in a prepared statement. “For this approval, we employed some of our newer regulatory tools to streamline reviews without compromising the quality of our assessment. This drug is the first novel drug approved under the Real-Time Oncology Review pilot program. We also used the updated Assessment Aid, a multidisciplinary review template that helps focus our written review on critical thinking and consistency and reduces time spent on administrative tasks.”

According to the FDA, the efficacy of alpelisib was studied in the SOLAR-1 trial, a randomized trial of 572 men and postmenopausal women with HR-positive, HER2-negative, advanced or metastatic breast cancer whose cancer had progressed while on or after receiving an aromatase inhibitor. Results from the trial 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.

Adverse events associated with alpelisib tablets include 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, aPTT prolonged, and hair loss.

Reference

FDA approves first PI3K inhibitor for breast cancer [news release]. Silver Spring, MD; May 24, 2019: FDA website. https://www.fda.gov/news-events/press-announcements/fda-approves-first-pi3k-inhibitor-breast-cancer. Accessed May 24, 2019.