NEW YORK – The European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP) on Friday recommended approval of alpelisib (Novartis' Piqray) in combination with fulvestrant for PIK3CA-mutated, hormone receptor-positive, HER2-negative, locally advanced or metastatic breast cancer that has progressed on endocrine therapy. The drug is recommended for women who are postmenopausal and men with breast cancer.
Approximately 40 percent of hormone receptor-positive, HER2-negative, advance breast cancers have a PIK3CA mutation. CHMP recommended the drug for approval based on results of the Phase III SOLAR-1 trial, in which PIK3CA-mutated breast cancer patients had a median progression-free survival of 11 months on alpelisib and fulvestrant compared to 5.7 months with fulvestrant alone. Even those with lung and liver metastases benefitted more from the alpelisib-containing regimen compared to just fulvestrant. The drug also had a mild-to-moderate toxicity profile, and most adverse events were manageable.
The European Commission will review CHMP's recommendation and is expected to issue a final decision in the next two months. If approved, the drug will be available in 27 European Union member states, the United Kingdom, Iceland, Norway, and Liechtenstein.
Data from SOLAR-1 supported approval of this same indication by the US Food and Drug Administration in March 2019. Simultaneously, the agency also approved Qiagen's Therascreen PIK3CA RGQ PCR Kit as a companion diagnostic for identifying which advanced breast cancer patients have PIK3CA mutations and are likely to respond to alpelisib. The test is designed to analyze PIK3CA mutations in tissue and blood samples. Patients who are mutation negative by liquid biopsy analysis should undergo tumor biopsy for tissue analysis.