Alpelisib

ANTINEOPLASTIC AND IMMUNOMODULATING AGENTS

Key facts

Alpelisib is an onp drug catalogued in DrugCentral. It was first approved by the FDA in 2019. DrugCentral lists 9 molecular targets, including PIK3CA, PI4KB and PIK3CG. Recorded indications include Germline BRCA-mutated, HER2-negative metastatic breast cancer and PIK3CA-related overgrowth spectrum (PROS).

• Approval status: onp (first approved 2019 by the FDA).
• Molecular targets: PIK3CA, PI4KB, PIK3CG and PIK3CD (9 listed in DrugCentral).
• Indications: Germline BRCA-mutated, HER2-negative metastatic breast cancer and PIK3CA-related overgrowth spectrum (PROS).
• ATC code: L01EM03 (Phosphatidylinositol-3-kinase (Pi3K) inhibitors).
• Molecular formula: C19H22F3N5O2S; molecular weight 441.47 g/mol.

Indications & contraindications

Drug–disease relationships curated by DrugCentral (OMOP / SNOMED concepts).

Indications (2)

• Germline BRCA-mutated, HER2-negative metastatic breast cancer
• PIK3CA-related overgrowth spectrum (PROS)

Mechanism & targets

Molecular targets (9)

Target	Gene	Action	Activity
Phosphatidylinositol 4,5-bisphosphate 3-kinase catalytic subunit alpha isoform	PIK3CA	inhibitor	IC50 8.34
Phosphatidylinositol 4-kinase beta	PI4KB	unknown	IC50 7.41
Phosphatidylinositol 4,5-bisphosphate 3-kinase catalytic subunit gamma isoform	PIK3CG	unknown	IC50 6.60
Phosphatidylinositol 4,5-bisphosphate 3-kinase catalytic subunit delta isoform	PIK3CD	unknown	IC50 6.54
Phosphatidylinositol 4,5-bisphosphate 3-kinase catalytic subunit beta isoform	PIK3CB	unknown	IC50 5.92
cAMP-dependent protein kinase catalytic subunit gamma	PRKACG	unknown	Kd 5.70
Phosphatidylinositol 4,5-bisphosphate 3-kinase catalytic subunit beta isoform	Pik3cb	unknown	IC50 5.66
Serine/threonine-protein kinase mTOR	MTOR	unknown	IC50 5.52
Cyclin-G-associated kinase	GAK	unknown	Kd 5.30

Regulatory approvals (2)

Date	Agency	Marketed as	Applicant	Orphan
2020-07-27	EMA	—	Novartis Europharm Limited
2019-04-24	FDA	—	NOVARTIS PHARMS CORP

ATC classification

ATC code	Therapeutic hierarchy
L01EM03	ANTINEOPLASTIC AND IMMUNOMODULATING AGENTS › ANTINEOPLASTIC AGENTS › PROTEIN KINASE INHIBITORS › Phosphatidylinositol-3-kinase (Pi3K) inhibitors

Pharmacogenomics

Drug-label and guideline annotations from PharmGKB and CPIC.

• EMA label: ERBB2, ESR1, ESR2, PIK3CA · Testing Required
• FDA label: ERBB2, ESR1, ESR2, PIK3CA · Testing Required
• HCSC label: ERBB2, ESR1, ESR2, PIK3CA · Testing Required

Reported adverse-event signals

Strongest FDA FAERS disproportionality signals (log-likelihood ratio). A signal is a statistical association in spontaneous reports, not proof that the drug causes the event.

MedDRA term	LLR
Hyperglycaemia	3843.6
Malignant neoplasm progression	846.3
Rash	820.2
Blood glucose increased	719.3
Metastases to bone	330.9
Metastases to liver	324.3
Death	309.3
Diabetic ketoacidosis	277.7
Diarrhoea	266.9
Diabetes mellitus	209.1
PIK3CA-activated mutation	149.8
Blood glucose abnormal	135.7

Chemical structure

Full physicochemical, ADMET and 3D-structure analysis of Alpelisib is on its molecule page.

• Molecular formula: C₁₉H₂₂F₃N₅O₂S
• Molecular weight: 441.47 g/mol
• CAS number: 1217486-61-7
• InChIKey: STUWGJZDJHPWGZ-LBPRGKRZSA-N
• PubChem CID: 56649450

CC1=C(SC(NC(=O)N2CCC[C@H]2C(N)=O)=N1)C1=CC(=NC=C1)C(C)(C)C(F)(F)F

Frequently asked questions

Answers are generated from the structured data on this page, not written by hand.

What is Alpelisib used for?

DrugCentral lists the following indications for Alpelisib: Germline BRCA-mutated, HER2-negative metastatic breast cancer and PIK3CA-related overgrowth spectrum (PROS).

How does Alpelisib work?

Its molecular targets in DrugCentral include PIK3CA (inhibitor), PI4KB, PIK3CG and PIK3CD.

When was Alpelisib first approved?

Alpelisib was first approved by the FDA in 2019.

What is the chemical formula of Alpelisib?

Alpelisib has the molecular formula C19H22F3N5O2S and a molecular weight of 441.47 g/mol.

Sources

• DrugCentral drug card 5328 — structure, indications, targets, approvals, pharmacologic classes (CC BY-SA 4.0)
• ChEMBL CHEMBL2396661 — compound record and bioactivity
• PubChem CID 56649450 — chemical identifiers
• DrugBank DB12015 — drug record
• FDA UNII 08W5N2C97Q — substance identity
• DailyMed: FDA labels for Alpelisib — current prescribing information (label search)
• UniProt P42336 (PIK3CA) — target protein
• UniProt Q9UBF8 (PI4KB) — target protein
• UniProt P48736 (PIK3CG) — target protein
• UniProt O00329 (PIK3CD) — target protein
• UniProt P42338 (PIK3CB) — target protein
• UniProt P22612 (PRKACG) — target protein
• FDA Adverse Event Reporting System (openFDA FAERS) — adverse-event signals, as aggregated by DrugCentral
• PharmGKB / CPIC — pharmacogenomic label annotations

How this page is built: methodology · editorial policy & corrections.