Drug · Small Molecule

Alpelisib

CI-DRUG-00000050Explore in graph →NCIt C94214 CHEMBL2396661 CIViC Approved

Regulatory

Approvals (12)

Each record names the authority, jurisdiction, indication text and status. A drug approved in one jurisdiction for one indication is not 'approved' in general.

openfdaRegulatory6 approval records · authority, jurisdiction and indication text as published.

Jurisdiction · authority	Cancer	Indication	Status	Approval date	Source
US FDA	—	Efficacy supplement 2024-04-24 (see label)	approved	Apr 24, 2024	openfda
US FDA	—	VIJOICE is indicated for the treatment of adult and pediatric patients 2 years of age and older with severe manifestations of PIK3CA-Related Overgrowth Spectrum (PROS) who require systemic therapy. This indication is approved under accelerated approval based on response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s). VIJOICE is a kinase inhibitor indicated for the treatment of adult and pediatric patients 2 years of age and older with severe manifestations of PIK3CA-Related Overgrowth Spectrum (PROS) who require systemic therapy. This indication is approved under accelerated approval based on response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s). ( 1 )accelerated	approved	Apr 24, 2024	openfda
US FDA	—	Efficacy supplement 2024-01-18 (see label)	approved	Jan 18, 2024	openfda

Health Canada records are DIN-level: one row per marketed product (brand, strength, form). The Drug Product Database does not publish indications, so no cancer is stated for these rows, and a cancelled or dormant DIN is the status of that one product — not a withdrawal of the molecule.

Data updated 11 hours agoSource updated unknown

Derived

Development pipeline

Most advanced stage across all cancers, then per top-level cancer reached through trial conditions or approval indications. Approval in any ingested jurisdiction outranks trial phase; counts are interventional studies.

Scope	Stage	Max phase	Active	Recruiting	Phase 3	Trials	Approvals	Jurisdictions	First approval	First trial
All cancers	Approved	Phase 4	23	6	10	76	12	CA, EU, US	May 24, 2019	Oct 5, 2010
Malignant Breast Neoplasm	Approved	Phase 4	15	3	7	36	2	EU, US	May 24, 2019	2011-08
Malignant Ovarian Neoplasm	Phase3	Phase 3	1	1	1	3	0	—	—	Jul 22, 2021
Malignant Uterine Corpus Neoplasm	Phase2	Phase 2	2	1	0	2

Curated evidence

Clinical evidence (22)

CIViC items in which this therapy appears, grouped by cancer context, then molecular profile. 50 items per page.

civicCuratedShowing 1–22 of 22 evidence items · levels, directions and significance as curated at the source; each row links to its CIViC record.

Therapy	Cancer	Type	Level	Direction · significance	Rating (1–5)	Status	Evidence	Source
PIK3CA Amplification1
Alpelisib	Gastric Carcinoma

Clinical trials

Trials with this intervention (81)

Most recently updated first, 50 per page.

clinicaltrialsPublishedShowing 1–50 of 81 studies · status and phase as posted by the registrant; each row links to the study record.

NCT	Title	Status	Phase	Enrollment (n)	Sponsor	Countries	Last update	Source
NCT05038735	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.(EPIK-B5)	active not recruiting	Phase 3	210	Novartis PharmaceuticalsINDUSTRY	17	Sep 29, 2026	clinicaltrials
NCT05564377	Targeted Therapy Directed by Genetic Testing in Treating Patients With Locally Advanced or Advanced Solid Tumors, The ComboMATCH Screening Trial

Molecule

Record

Kind: Small Molecule
Mechanism: PI3-kinase p110-alpha subunit inhibitor
Targets: 1 gene
UNII: 08W5N2C97Q
Aliases: Alpelisib, ALPELISIB (BYL719), BYL 719, BYL719, BYL-719A-1214(S)-N1-(4-METHYL-5-(2-(1,1,1-TRIFLUORO-2-METHYLPROPAN-2-YL)PYRIDIN-4-YL)THIAZOL-2-YL)PYRROLIDINE-1,2-DICARBOXAMIDE, NVP-BYL719, Phosphoinositide 3-kinase Inhibitor BYL719, Piqray, PIQRAY, VIJOICE

Data updated 14 hours agoSource updated unknown

Identifiers

Codes (12)

Upstream identifiers kept as first-class codes; each links to its registry where a stable public URL exists.

System	Code	Label
ATC (WHO)	L01EM03	ALPELISIB
ATC (WHO)	L01XE	Antineoplastic agents
DIN (Health Canada)

HRAS G12V1
Alpelisib	Head and Neck Squamous Cell Carcinoma	Predictive	D	Supports Resistance	1	submitted	EID9087 In this preclinical study, cell lines with constitutively-active G12V mutant found that forced overexpression of HRAS G12V caused a reduced susceptibility to PI3K inhibitor BYL719 in comparison to wil… (full text at CIViC) PMID 30115483 · Ruicci et al., 2018 · Open in CIViC	civic

HRAS G12V1

Alpelisib

Head and Neck Squamous Cell Carcinoma

Predictive

Supports Resistance

submitted

EID9087

In this preclinical study, cell lines with constitutively-active G12V mutant found that forced overexpression of HRAS G12V caused a reduced susceptibility to PI3K inhibitor BYL719 in comparison to wil… (full text at CIViC)

PMID 30115483 · Ruicci et al., 2018 · Open in CIViC

PIK3CA Mutation3
Alpelisib + FulvestrantCombination	Malignant Breast Neoplasm	Predictive	A	Supports Sensitivity Response	5	accepted	EID7313 In a phase 3 trial, patients with hormone receptor positive, HER2 receptor negative breast were allocated to alpelisib plus fulvestrant or placebo plus fulvestrant. Patients were enrolled into two coh… (full text at CIViC) PMID 31091374 · André et al., 2019 · Open in CIViC	civic
〃	Malignant Breast Neoplasm	Predictive	A	Supports Sensitivity Response	5	rejected	EID7317 In a phase 3 trial, patients with hormone receptor positive, HER2 receptor negative breast were allocated to alpelisib plus fulvestrant or placebo plus fulvestrant. Patients were enrolled into two coh… (full text at CIViC) PMID 31091374 · André et al., 2019 · Open in CIViC	civic
Alpelisib	Malignant NeoplasmALIAS	Predictive	D	Supports Sensitivity Response	4	accepted	EID1402 A set of 474 cell lines from the Cancer Cell Line Encyclopedia was tested for responsiveness to the PI3Kalpha specific inhibitor BYL719 (Alpelisib). In the PIK3CA wild type subroup of cell lines, 22% … (full text at CIViC) PMID 24608574 · Fritsch et al., 2014 · Open in CIViC	civic