What are the specific indications for Alpelisib?

Alpelisib(Alpelisib) is a targeted drug developed by the Swiss pharmaceutical company Novartis Ltd. (Novartis Ltd). As a PI3K inhibitor, Alpelisib is mainly used to treat specific types of cancer, especially advanced or metastatic breast cancer. Its mechanism of action is to block the growth and proliferation of cancer cells by inhibiting the PI3K signaling pathway, thereby delaying the progression of the disease. The indications of Apelvis cover two main clinical applications, namely the treatment of advanced breast cancer and PIK3CA-related overgrowth spectrum (PROS).

First, Apelvis (Piqray brand) is indicated in combination with fulvestrant to treat postmenopausal women and men with advanced or metastatic breast cancer. These patients must meet specific criteria: the cancer needs to be hormone receptor (HR) positive, human epidermal growth factor receptor 2 (HER2) negative, and have a PIK3CA gene mutation. Patients with this type of breast cancer are often resistant to endocrine-based treatments (such as tamoxifen or anastrozole). Therefore, the combination of apelvis and fulvestrant can effectively inhibit the PI3K signaling pathway and improve the efficacy. With this combination treatment, patients can delay disease progression, achieve longer progression-free survival (PFS) and better quality of life.

Secondly, Apelvis (Vijoice brand) is also approved to treat patients 2 years and older with PIK3CA-associated overgrowth spectrum (PROS). PROS is a rare genetic disease that usually manifests as overgrowth or lumps in certain parts of the body. Patients often experience symptoms such as abnormal skin, soft tissue or bone growth. When treating such patients, Apelvis inhibits abnormal cell proliferation by targeting PIK3CA mutations, helping to slow down the progression of the disease and improve the patient's symptoms. Approval of this indication provides a new treatment option for patients with severe manifestations of PROS, especially in adult and pediatric patients who require systemic therapy.

Reference materials:https://www.ema.europa.eu/en/documents/product-information/piqray-epar-product-information_en.pdf