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Drug

Alpelisib

Approved · FDA / EMA
ClassPI3-kinase p110-alpha subunit inhibitorEmerging researchLate-stage developmentSteady momentum

Also known as Piqray, Vijoice.

RxNorm2169285ChEMBLCHEMBL2396661ATCL01EM03UNII08W5N2C97Q
4
Research papers
29
Active clinical trials
PIK3CA
Primary target
6
Regulatory events
Latest activity
beta

Recent clinical, regulatory, research and industry developments relating to this drug.

A Two-stage Double-blind, Randomized, Placebo-controlled Study to Assess the Efficacy, Safety and Pharmacokinetics of Alpelisib in Pediatric and Adult Patients With Lymphatic Malformations Associated With a PIK3CA Mutation.

Clinical trial2026-07-14Results expected Q2 2028 · ClinicalTrials.gov

ALPelisib INdia Safety STudy (ALPINIST): A Phase IV, Prospective, Multicenter, Open-label, Non-comparative, Interventional Study to Assess the Safety of Alpelisib Plus Fulvestrant, in Men and Post-menopausal Women With HR Positive, HER2-negative, Advanced Breast Cancer (aBC) With a PIK3CA Mutation, Whose Disease Has Progressed on or After Endocrine Based Treatment.

Clinical trial2026-07-09Results posted · ClinicalTrials.gov

Molecular Analysis for Combination Therapy Choice (ComboMATCH)

Clinical trial2026-07-09Results expected Q3 2030 · ClinicalTrials.gov

EPIK-B5: A Phase III, Randomized, Double-blind, Placebo-controlled Study of Alpelisib in Combination With Fulvestrant for Men and Postmenopausal Women With HR-positive, HER2-negative Advanced Breast Cancer With a PIK3CA Mutation, Who Progressed on or After Aromatase Inhibitor and a CDK4/6 Inhibitor

Clinical trial2026-07-08Results expected Q1 2027 · ClinicalTrials.gov

CHMP positive opinion: Vijoice (EMA)

Regulatory2026-05-22EMA

Label change: ALPELISIB (NDA215039)

Regulatory2025-07-22FDA

Indication expansion: ALPELISIB (NDA215039)

Regulatory2024-04-24FDA

Incidence, risk factors, and management of alpelisib-associated hyperglycemia in metastatic breast cancer.

Research2023-09-25Cancer

Label change: ALPELISIB (NDA215039)

Regulatory2022-11-17FDA

Alpelisib plus fulvestrant for PIK3CA-mutated, hormone receptor-positive, human epidermal growth factor receptor-2-negative advanced breast cancer: final overall survival results from SOLAR-1.

Research2020-11-25Annals of oncology : official journal of the European Society for Medical Oncology

Frequency and spectrum of PIK3CA somatic mutations in breast cancer.

Research2020-05-13Breast cancer research : BCR

Alpelisib for <i>PIK3CA</i>-Mutated, Hormone Receptor-Positive Advanced Breast Cancer.

Research2019-05-01The New England journal of medicine

Profile

Identifiers & mechanism

Canonical identifiers, marketed brand names and mechanism, resolved across RxNorm, ChEMBL and ATC.

Canonical name
Alpelisib
Aliases & brands
PiqrayVijoice
RxNorm CUI
2169285
ChEMBL ID
CHEMBL2396661
ATC codes
L01EM03
UNII
08W5N2C97Q
Primary mechanism
PI3-kinase p110-alpha subunit inhibitor
Regulatory jurisdictions
fdaema

Pharmacology & targets

1 target

Known molecular targets and mechanisms supported by curated pharmacology databases.

Phosphatidylinositol 4,5-bisphosphate 3-kinase catalytic subunit alpha isoform
PIK3CA · P42336
INHIBITORPI3-kinase p110-alpha subunit inhibitor

Regulatory timeline

6 events

The complete regulatory record, grouped by authority — approvals, safety advisories and label changes. Each authority shows its most recent events; expand one to read its full history.

Approval / market entry confirmed across FDA and EMA (2020-07-27 → 2022-04-05)
Earliest approval
2020-07-27
Latest approval
2024-04-24
Authorities
FDA · EMA
Total events
6
fdaU.S. Food and Drug Administration· 4 events
2025-07-22Label change
Label change: ALPELISIB (NDA215039)
Evidence ↗
2024-04-24Indication expansion
Indication expansion: ALPELISIB (NDA215039)
Evidence ↗
2022-11-17Label change
Label change: ALPELISIB (NDA215039)
Evidence ↗
emaEuropean Medicines Agency· 2 events
2026-05-22CHMP positive opinion
CHMP positive opinion: Vijoice (EMA)

Indication: Treatment of adult and paediatric patients aged 2 years and older with severe or life-threatening manifestations of PIK3CA-related overgrowth spectrum (PROS)

Evidence ↗
2020-07-27ApprovalMulti-authority
Approval: Piqray (EMA)
Indication: 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… Show full indication

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 (see section 5.1).

Evidence ↗

Data from the U.S. Food and Drug Administration (U.S. Food and Drug Administration), public domain (CC0).

Contains information from the European Medicines Agency (European Medicines Agency), © EMA, reused under CC BY 4.0.

Clinical trials

56 trials

The current development programme across all trial phases — status mix, phase distribution and the late-stage studies shaping the evidence base.

Development programme
CLINICALTRIALS.GOV · LIVE REGISTRY
56
registered trials across all phases
LATEST COMPLETION 2025
29
Active studies
11
Recruiting
12
Late-stage (III+)
11
Completed
16
Discontinued
PHASE DISTRIBUTIONn = 56
Early Phase 11Phase 111Phase 1 / 25Phase 227Phase 2 / 31Phase 39Phase 41Phase N / A1

Late-stage studies

Phase III+ trials still open or recently active — where late-stage evidence is being generated.

ALPelisib INdia Safety STudy (ALPINIST): A Phase IV, Prospective, Multicenter, Open-label, Non-comparative, Interventional Study to Assess the Safety of Alpelisib Plus Fulvestrant, in Men and Post-menopausal Women With HR Positive, HER2-negative, Advanced Breast Cancer (aBC) With a PIK3CA Mutation, Whose Disease Has Progressed on or After Endocrine Based Treatment.

CompletedPH IVNovartis Pharmaceuticalsn=402025

A Two-stage Double-blind, Randomized, Placebo-controlled Study to Assess the Efficacy, Safety and Pharmacokinetics of Alpelisib in Pediatric and Adult Patients With Lymphatic Malformations Associated With a PIK3CA Mutation.

RecruitingPH II/IIINovartis Pharmaceuticalsn=2322033

A Phase III, Multicenter, Randomized, Open-Label Study Evaluating the Efficacy and Safety of Inavolisib Plus Fulvestrant Versus Alpelisib Plus Fulvestrant in Patients With Hormone Receptor-Positive, HER2-Negative, PIK3CA Mutated, Locally Advanced or Metastatic Breast Cancer Who Progressed During or After CDK4/6 Inhibitor and Endocrine Combination Therapy

Active, not recruitingPH IIIHoffmann-La Rochen=4202029

EPIK-B5: A Phase III, Randomized, Double-blind, Placebo-controlled Study of Alpelisib in Combination With Fulvestrant for Men and Postmenopausal Women With HR-positive, HER2-negative Advanced Breast Cancer With a PIK3CA Mutation, Who Progressed on or After Aromatase Inhibitor and a CDK4/6 Inhibitor

Active, not recruitingPH IIINovartis Pharmaceuticalsn=2102027

EPIK-B2: A Two Part, Phase III, Multicenter, Randomized (1:1), Double-blind, Placebo-controlled Study to Assess the Efficacy and Safety of Alpelisib (BYL719) in Combination With Trastuzumab and Pertuzumab as Maintenance Therapy in Patients With HER2-positive Advanced Breast Cancer With a PIK3CA Mutation

Active, not recruitingPH IIINovartis Pharmaceuticalsn=192027

Recent completions

Trials that read out recently, adding to the completed evidence base.

A Phase 1/2 Open-label, Biomarker-defined Cohort Trial to Evaluate the Safety, Determine the Recommended Combination Dosing, and Assess Early Antitumor Activity of Tipifarnib and Alpelisib for the Treatment of Adult Participants Who Have HRAS-overexpressing and/or PIK3CA-mutated and/or - Amplified Recurrent/Metastatic Head and Neck Squamous Cell Carcinoma

CompletedPH I/IIKura Oncology, Inc.n=452025

ALPelisib INdia Safety STudy (ALPINIST): A Phase IV, Prospective, Multicenter, Open-label, Non-comparative, Interventional Study to Assess the Safety of Alpelisib Plus Fulvestrant, in Men and Post-menopausal Women With HR Positive, HER2-negative, Advanced Breast Cancer (aBC) With a PIK3CA Mutation, Whose Disease Has Progressed on or After Endocrine Based Treatment.

CompletedPH IVNovartis Pharmaceuticalsn=402025

Study to Evaluate the Effect of Metformin in the Prevention of Hyperglycemia in HR[+]/HER2[-] PIK3CA-mutation Advanced Breast Cancer Patients Treated With Alpelisib Plus Endocrine Therapy. Study Metallica

CompletedPH IIMedSIRn=692025

BYLieve: A Phase II, Multicenter, Open-label, Three-cohort, Non- Comparative Study to Assess the Efficacy and Safety of Alpelisib Plus Fulvestrant or Letrozole in Patients With PIK3CA Mutant, Hormone Receptor (HR) Positive, HER2-negative Advanced Breast Cancer (aBC), Who Have Progressed on or After Prior Treatments

CompletedPH IINovartis Pharmaceuticalsn=3832024

An Umbrella, Randomized, Controlled, Pre Operative Trial Testing Integrative Subtype Targeted Therapeutics in Estrogen Receptor Positive, HER2-Negative Breast Cancer

CompletedPH IIJennifer Lee Caswell-Jinn=192024

Phase Ib Study of BYL719 (Alpelisib) in Combination With Androgen Receptor Inhibitor (Enzalutamide) in Patients With Androgen Receptor (AR)-Positive and PTEN Positive Metastatic Breast Cancer

CompletedPH IM.D. Anderson Cancer Centern=182023

Research activity

4 papers

Key research shaping understanding of this drug, combining the latest publications with the most influential evidence.

Publications over time
20192023
Major research themes4
Breast Neoplasms2Antineoplastic Combined Chemotherapy Protocols1Hyperglycemia1Mutation1
Journals, researchers & institutions
Top journals
  • Annals of oncology : official journal of the European Society for Medical Oncology1
  • Breast cancer research : BCR1
  • Cancer1
  • The New England journal of medicine1
Leading researchers
  • André F2
  • Campone M2
  • Inoue K2
  • Iwata H2
  • Juric D2
  • Kaufman B2
  • Loibl S2
  • Lu YS2
Leading institutions
free-text, unnormalised
  • Aichi Cancer Center Hospital1
  • Department of Medical Oncology and Clinical Pharmacology1
  • From Institut Gustave Roussy1
  • Hospital Clinic of Barcelona1
  • Hospital Universitario 12 de Octubre1
  • Hungarian Defence Forces Medical Centre1

Related diseases

1 conditions

Regulator-approved indications and conditions co-mentioned with this drug across the research literature. Number shows shared papers.

Active research areas
Breast Neoplasms4

Related drugs

1 matches

Drugs sharing diseases, protein targets and literature with this one. Ranked by graph evidence (shared targets + diseases weighted above co-mentions).

Fulvestrant

Studied across 2 of the same disease areas as Alpelisib in the shared literature.

2 shared diseases2 shared papers
References & data sources
  • RxNorm (U.S. National Library of Medicine) — drug identity
  • ChEMBL (EMBL-EBI) & UniProt — pharmacology and targets
  • Europe PMC — research literature
  • ClinicalTrials.gov — clinical trials
  • Regulatory event sources are credited in the Regulatory Timeline above.