FDA Approves Novartis’ PI3K Inhibitor for Breast Cancer

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The agency says Novartis drug is the first PI3K inhibitor to be approved for treating breast cancer.

On May 24, 2019, FDA granted Novartis approval for Piqray (alpelisib) tablets, a phosphoinositide 3-kinase (PI3K) inhibitor to be used in combination with the FDA-approved endocrine therapy fulvestrant to treat breast cancer. The drug is indicated for 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.

In conjunction with the Piqray approval, FDA also approved the companion diagnostic test, therascreen PIK3CA RGQ PCR Kit (QIAGEN Manchester), 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 would need to undergo tumor biopsy for PIK3CA mutation testing, according to the agency.

FDA notes that the drug is the first PI3K inhibitor approved 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 FDA’s Oncology Center of Excellence and acting director of the Office of Hematology and Oncology Products in FDA’s Center for Drug Evaluation and Research, in an agency press release.

“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,” Pazdur added.


The Real-Time Oncology Review (RTOR) pilot program permits FDA to begin analyzing key efficacy and safety datasets prior to the official submission of a new drug application, allowing the review team to begin their review and communicate with the applicant earlier. Piqray also used the updated Assessment Aid (AAid), a multidisciplinary review template intended to focus FDA’s written review on critical thinking and consistency and reduce time spent on administrative tasks. With these two pilot programs, the Piqray approval comes approximately three months ahead of the Prescription Drug User Fee Act (PDUFA) VI deadline of Aug. 18, 2019, according to the agency.

Metastatic breast cancer is breast cancer that has spread beyond the breast to other organs in the body (most often the bones, lungs, liver, or brain). When breast cancer is hormone-receptor positive, patients may be treated with anti-hormonal treatment (endocrine therapy) alone or in combination with other medicines, or chemotherapy.

Source: FDA