The Food and Drug Administration (FDA) has approved Piqray (alpelisib; Novartis) in combination with fulvestrant for the treatment of postmenopausal women, and men, with hormone receptor (HR)-positive, human epidermal growth factor receptor 2 (HER2)-negative, PIK3CA-mutated, advanced or metastatic breast cancer as detected by an FDA-approved test following progression on or after an endocrine-based regimen.

Alpelisib is an inhibitor of phosphatidylinositol-3-kinase (PI3K). In breast cancer cell lines, alpelisib inhibited the phosphorylation of PI3K downstream targets, including Akt and showed activity in cell lines harboring a PIK3CA mutation; PIK3CA mutations are present in approximately 40% of HR-positive advanced or metastatic breast cancer patients.

The approval was based on data from the SOLAR-1 trial in which 572 patients with HR-positive, HER2-negative, advanced or metastatic breast cancer whose disease had progressed or recurred on or after an aromatase inhibitor-based treatment (with or without CDK4/6 combination) were randomized to receive Piqray plus fulvestrant or placebo plus fulvestrant. The primary endpoint of the study was investigator-assessed progression-free survival (PFS) in the cohort with a PIK3CA mutation (n=341).

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Among patients with a PIK3CA mutation treated with Piqray plus fulvestrant, median PFS was 11.0 months, while in those treated with placebo plus fulvestrant, median PFS was 5.7 months (hazard ratio [HR] 0.65; 95% CI: 0.50-0.85; P =.0013); no PFS benefit was observed in patients whose tumors did not have a PIK3CA tissue mutation (HR=0.85; 95% CI: 0.58-1.25). In addition, overall response rate, defined as the proportion of patients who experienced at least a 30% reduction in overall tumor size, was found to be greater in the combination therapy group vs the placebo plus fulvestrant group (35.7% vs 16.2%; P =.0002).

“In the SOLAR-1 phase 3 trial, alpelisib plus fulvestrant nearly doubled median PFS and more than doubled overall response rate in patients with a PIK3CA mutation, offering them new hope for longer life without progression,” said Fabrice André, MD, PhD, research director and head of INSERM Unit U981, professor in the Department of Medical Oncology at Institut Gustave Roussy in Villejuif, France, and global SOLAR-1 principal investigator. “For the first time, physicians can test for PIK3CA biomarkers and develop a treatment plan based on the genomic profile of a patient’s cancer.”

As for safety, the most common adverse reactions observed in the study included increased glucose, increased creatinine, diarrhea, rash, decreased lymphocyte count, increased GGT, nausea, increased ALT, fatigue, decreased hemoglobin, increased lipase, decreased appetite, stomatitis, vomiting, decreased weight, decreased calcium, decreased glucose, prolonged aPTT, and alopecia.

Piqray will be available in 50mg, 150mg, and 200mg tablets.

For more information visit novartis.com.

This article originally appeared on MPR