Alpelisib

Alpelisib is a targeted small-molecule therapy developed for the treatment of hormone receptor–positive breast cancer driven by specific genetic alterations. It represents an important advance in precision oncology by focusing on a defined molecular pathway known to contribute to cancer growth and treatment resistance.

At the molecular level, Alpelisib is a selective inhibitor of phosphatidylinositol 3-kinase alpha (PI3Kα). This enzyme plays a central role in the PI3K/AKT signaling pathway, which regulates cell growth, metabolism, and survival. In cancers with activating PIK3CA mutations, this pathway becomes overactive. By selectively inhibiting PI3Kα, Alpelisib helps suppress tumor cell proliferation while minimizing activity against other PI3K isoforms.

Because of its targeted mechanism and use in combination therapy, molecular purity, stability, and consistency are essential to ensure predictable clinical performance. Alpelisib has become a key molecule in the management of genetically defined breast cancer.

Prescribing Information

Prescribing information provides official guidance on approved indications, dosing, administration, contraindications, and safety monitoring.

Clinical Trial Details

The clinical efficacy and safety of Alpelisib were established through pivotal clinical studies, most notably the SOLAR-1 trial. This Phase III study demonstrated improved progression-free survival in patients with HR-positive, HER2-negative, PIK3CA-mutated advanced breast cancer when Alpelisib was used in combination with endocrine therapy.

FDA Approval Status

Alpelisib is approved by the U.S. Food and Drug Administration for use in combination with endocrine therapy for patients with HR-positive, HER2-negative, PIK3CA-mutated advanced or metastatic breast cancer. It is marketed under the brand name Piqray® by Novartis.