Regulatory approval
Published by the European Medicines Agency.
The European Medicines Agency (EMA) has authorized alpelisib in combination with fulvestrant for the treatment of patients who are either postmenopausal women or men with hormone receptor (HR)-positive, human epidermal growth factor receptor 2 (HER2)-negative, locally advanced or metastatic breast cancer with a PIK3CA mutation after disease progression following endocrine therapy as a monotherapy. This indication is based on C2301, a phase 3, randomized, double-blind, and placebo-controlled study that evaluated alpelisib in combination with fulvestrant based on the presence or absence of a PIK3CA. No survival benefit was shown in the group without PIK3CA variants and PIK3CA variants observed were: p.C420R, p.E542K, p.E545A, p.E545D (1635G>T), p.E545G, p.E545K, p.Q546E, p.Q546R, p.H1047L, p.H1047R, and p.H1047Y.
This is written in the approval document as:
Piqray is indicated in combination with fulvestrant for the treatment of postmenopausal women, and men, with hormone receptor (HR)-positive, human epidermal growth factor receptor 2 (HER2)-negative, locally advanced or metastatic breast cancer with a PIK3CA mutation after disease progression following endocrine therapy as monotherapy.
Citation
Novartis Europharm Limited. Piqray (alpelisib) [product information]. European Medicines Agency website. https://www.ema.europa.eu/en/documents/product-information/piqray-epar-product-information_en.pdf. Revised October 2023. Accessed March 22, 2024.Therapeutic response
Precision oncology relationships for therapeutic response derived from this regulatory approval.| Type | Biomarker(s) | Cancer type | Therapy(ies) | |
|---|---|---|---|---|
| Sensitivity (+) | ER positive, HER2-negative, PIK3CA somatic variants | Invasive Breast Carcinoma | Alpelisib, Fulvestrant | |
| Sensitivity (+) | HER2-negative, PIK3CA somatic variants, PR positive | Invasive Breast Carcinoma | Alpelisib, Fulvestrant | |
| Sensitivity (+) | ER positive, HER2-negative, PIK3CA somatic variants, PR positive | Invasive Breast Carcinoma | Alpelisib, Fulvestrant |