# Alpelisib

Source: https://onco.cc/drugs/alpelisib/  
OnCo record `alpelisib` (Treatment). Data CC BY-NC 4.0, attribute "Data from OnCo (onco.cc)"; commercial use needs a licence.

## TL;DR

Alpelisib was the first PI3K drug for PIK3CA-mutant breast cancer (2019). It is effective, but high blood sugar and rash limited its use, and newer drugs are displacing it.

## Summary

SOLAR-1: PFS 11.0 vs 5.7 months with fulvestrant in PIK3CA-mutant disease. Hyperglycaemia (grade 3-4 ~37%), rash, and diarrhoea are frequent; requires metformin prophylaxis strategies. Superseded in first-line endocrine-resistant disease by inavolisib and in the unselected AKT-pathway-altered population by capivasertib.

## Fields

- Kind: Treatment
- Status: approved
- Last checked: 2026-09-07
- Brand: Piqray
- Modality: Small-molecule PI3Kα inhibitor
- Mechanism: ATP-competitive PI3Kα-selective inhibitor.
- Approvals: US 2019: PIK3CA-mutant HR+/HER2- advanced breast cancer with fulvestrant after endocrine therapy
- Dosing: Oral; 300 mg once daily with fulvestrant
- Toxicity (grade 3+): Hyperglycaemia 37%; Diarrhoea 7%; Rash 10%

## Sources

- Wikipedia: https://en.wikipedia.org/wiki/Alpelisib
- FDA label (DailyMed): https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Alpelisib

## Connected records

- biomarkers: [ER status (oestrogen receptor by IHC)](https://onco.cc/biomarkers/er-status/), [PIK3CA mutation](https://onco.cc/biomarkers/pik3ca-hotspot-mutation/)
- cancers: [HR-positive / HER2-negative breast cancer](https://onco.cc/cancers/breast-hr-positive/), [HR-positive metastatic breast cancer after CDK4/6 inhibitors](https://onco.cc/cancers/hr-positive-metastatic-post-cdk46/)
- technologies: [PI3K, AKT and mTOR inhibitors](https://onco.cc/technologies/pi3k-akt-mtor-inhibitors/), [Small-molecule kinase inhibitors](https://onco.cc/technologies/kinase-inhibitors/)
- targets: [PIK3CA / PI3K-alpha](https://onco.cc/targets/pik3ca/)
- companies: [Novartis](https://onco.cc/companies/novartis/)
- pathways: [Breast cancer (KEGG map)](https://onco.cc/pathways/breast-cancer-signalling/), [Chemical carcinogenesis - receptor activation](https://onco.cc/pathways/chemical-carcinogenesis-receptor-activation/), [Choline metabolism in cancer](https://onco.cc/pathways/choline-metabolism-in-cancer/), [MicroRNAs in cancer](https://onco.cc/pathways/micrornas-in-cancer/), [PI3K / AKT / mTOR](https://onco.cc/pathways/pi3k-akt-mtor/)
- terms: [Hyperglycaemia (PI3K/AKT inhibitor class effect)](https://onco.cc/terms/hyperglycaemia/), [PTEN loss](https://onco.cc/terms/pten-loss/)
- trials: [A Study Evaluating the Efficacy and Safety of Inavolisib Plus Fulvestrant Compared With Alpelisib Plus Fulvestrant in Participants With HR-Positive, HER2-Negative, PIK3CA Mutated, Locally Advanced or Metastatic Breast Cancer Post CDK4/6i and Endocrine Combination Therapy](https://onco.cc/trials/nct05646862/), [Alpelisib in Pediatric and Adult Patients With Lymphatic Malformations Associated With a PIK3CA Mutation.](https://onco.cc/trials/nct05948943/), [Clinical Trial of Alpelisb and Tucatinib in Patients With PIK3CA-Mutant HER2+ Metastatic Breast Cancer.](https://onco.cc/trials/nct05230810/), [Gedatolisib Plus Fulvestrant With or Without Palbociclib vs Standard-of-Care for the Treatment of Patients With Advanced or Metastatic HR+/HER2- Breast Cancer (VIKTORIA-1)](https://onco.cc/trials/nct05501886/), [Open-Label Umbrella Study To Evaluate Safety And Efficacy Of Elacestrant In Various Combination In Participants With Metastatic Breast Cancer](https://onco.cc/trials/nct05563220/), [SOLAR-1](https://onco.cc/trials/solar-1/), [Study Assessing the Efficacy and Safety of Treatment With Alpelisib Plus Fulvestrant in Japanese Men and Postmenopausal Women With Advanced Breast Cancer](https://onco.cc/trials/nct04524000/), [Study Assessing the Efficacy and Safety of Treatment With Alpelisib Plus Fulvestrant Versus Placebo Plus Fulvestrant in Chinese Men and Postmenopausal Women With Advanced Breast Cancer](https://onco.cc/trials/nct04544189/), [Study of Alpelisib (BYL719) in Combination With Trastuzumab and Pertuzumab as Maintenance Therapy in Patients With HER2-positive Advanced Breast Cancer With a PIK3CA Mutation](https://onco.cc/trials/nct04208178/), [Study to Assess the Efficacy and Safety of Alpelisib Plus Fulvestrant in Participants With HR-positive (HR+), HER2-negative, Advanced Breast Cancer After Treatment With a CDK4/6 Inhibitor and an Aromatase Inhibitor.](https://onco.cc/trials/nct05038735/)
- pairings: [PI3K/AKT-pathway inhibitor + endocrine therapy (± CDK4/6)](https://onco.cc/pairings/pi3k-pathway-plus-endocrine/)
- people: [Fabrice André](https://onco.cc/people/fabrice-andre/), [Sherry Lansing](https://onco.cc/people/sherry-lansing/)
- key papers: [SOLAR-1: alpelisib plus fulvestrant for PIK3CA-mutated hormone receptor-positive advanced breast cancer](https://onco.cc/key-papers/paper-solar-1-alpelisib-nejm-2019/)
- drugs: [therascreen companion diagnostic kits (KRAS, EGFR, PIK3CA, FGFR, BRAF)](https://onco.cc/drugs/therascreen-cdx/)
- roadmaps: [Hormonal therapy roadmap: removing the ovaries → tamoxifen → oral degraders switched by a blood test](https://onco.cc/roadmaps/hormonal-therapy-roadmap/)

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