FDA Approves Alpelisib as First PI3K Inhibitor for Treatment of Breast Cancer

The FDA has approved alpelisib (Piqray; Novartis) for the treatment of postmenopausal patients with hormone receptor (HR)-positive, HER2-negative, PIK3CA-mutated, advanced or metastatic breast cancer that has been detected by an FDA-approved test and progressed during or after an endocrine-based therapy regimen, in combination with fulvestrant.

Approval was also granted by the FDA to therascreen PIK3CA RGQ PCR Kit, a companion diagnostic test for the detection of PIK3CA mutation via a tissue and/or liquid biopsy.

“Piqray is the first PI3K inhibitor to demonstrate a clinically meaningful benefit in treating patients with this type of breast cancer. 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,” said Richard Pazdur, MD, Director of the FDA’s Oncology Center of Excellence, in a press statement.

This approval for alpelisib was based on the SOLAR-1 clinical trial of 572 postmenopausal women and men with HR-positive, HER2-negative, advanced or metastatic breast cancer that had progressed during or after therapy with aromatase inhibitor. Findings from the randomized study demonstrated that adding alpelisib to fulvestrant therapy significantly prolonged progression-free survival in patients with the PIK3CA mutation.

Side effects frequently reported with 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, aPTT, and hair loss.

Of note, the application for this drug was evaluated through the Priority Review process. In addition, this is the first new drug application for a new molecular entity that has been approved as part of the Real-Time Oncology Review pilot program, which allows the FDA to assess key efficacy and safety datasets before an application has officially been submitted.—Hina Khaliq

Source: US Food and Drug Administration. FDA approves first PI3K inhibitor for breast cancer. May 24, 2019. https://www.fda.gov/news-events/press-announcements/fda-approves-first-pi3k-inhibitor-breast-cancer. Accessed May 28, 2019.