BRIEF—FDA approves Piqray, a new treatment for PIK3CA-mutated advanced breast cancer

On Friday, the US Food and Drug Administration approved Piqray (alpelisib; formerly BYL719) tablets, to be used in combination with the FDA-approved 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 (as detected by an FDA-approved test) following progression on or after an endocrine-based regimen.

With this news, Friday was a bonanza day for the drug’s developer, Swiss pharma giant Novartis, which also gained approval from the agency for its much anticipated  gene therapy Zolgensma (onasemnogene abeparvovec-xioi).

Until now, there were no approved PI3K inhibitors for HR+ advanced breast cancer and analysts have predicted big things for BYL719, with Baader Helvea’s analyst Bruno Bulic giving an 80% chance of sales reaching $1.9 billion per year.

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 are negative by the therascreen test using the liquid biopsy should undergo tumor biopsy for PIK3CA mutation testing.

Common side effects of Piqray 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 (enzymes released by the pancreas), decreased appetite, stomatitis, vomiting, weight loss, low calcium levels, aPTT prolonged (blood clotting taking longer to occur than it should), and hair loss