FDA approves PI3K inhibitor alpelisib for breast cancer


The Food and Drug Administration has approved the first PI3K inhibitor for the treatment of breast cancer.

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The drug, alpelisib (Piqray), was approved for use in combination with fulvestrant for men and postmenopausal women who have hormone receptor (HR)–positive, human epidermal growth factor receptor 2 (HER2)–negative, PIK3CA-mutated, advanced or metastatic breast cancer after progression on, or after, an endocrine-based regimen. The approval was announced by the FDA in a statement.

The agency also approved a diagnostic test – the therascreen PIK3CA RGQ PCR Kit – for detecting the PIK3CA mutation. The approval is based on results from the SOLAR-1 trial. In 572 HR-positive, HER2-negative patients with advanced or metastatic breast cancer who progressed after, or during, treatment with an aromatase inhibitor, the addition of alpelisib to fulvestrant significantly improved progression-free survival in patients with PIK3CA mutated tumors.

Median progression-free survival was 5.7 months on fulvestrant plus placebo, versus 11 months with fulvestrant plus alpelisib (hazard ratio for progression or death, 0.65; 95% confidence interval, 0.50-0.85; P less than .001). The results of the trial were recently published in the New England Journal of Medicine (2019 May 16;380[20]:1929-40).

In the FDA statement, Richard Pazdur, MD, director of the FDA’s Oncology Center of Excellence, noted that alpelisib is the “first PI3K inhibitor to demonstrate a clinically meaningful benefit in treating patients with this type of breast cancer.”

The drug’s application was approved under the Real-Time Oncology Review pilot program, which allows the FDA to begin analyzing efficacy and safety databases before a new drug application is even submitted.

The FDA noted that patients should not start treatment on alpelisib if they have a history of severe skin reactions, such as Stevens-Johnson syndrome, erythema multiforme, or toxic epidermal necrolysis.

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