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Alpelisib is a small molecule, orally bioavailable phosphatidylinositol 3-kinase (PI3K) inhibitor that selectively targets the p110α catalytic subunit of class I PI3K. By inhibiting this enzyme, which is frequently mutated in various cancers (notably breast cancer), alpelisib blocks the PI3K/AKT signaling pathway, reducing tumor cell growth and survival. It is primarily indicated for use in combination with fulvestrant to treat hormone receptor-positive (HR+), human epidermal growth factor receptor 2-negative (HER2−), PIK3CA-mutated advanced or metastatic breast cancer in adults whose disease has progressed on or after endocrine therapy. Alpelisib is also approved for treating severe manifestations of PIK3CA-related overgrowth spectrum (PROS) in adults and children aged two years and older who require systemic therapy[1][2][3][5][6].
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