avapritinib 🐶 Veterinary Use | Indications/Contra | FAERs-F | FAERs-M | Orange Bk | BioActivity |

Stem definition	Drug id	CAS RN
tyrosine kinase inhibitors	5379	1703793-34-3

Description:

Molecule	Description
Molfile Inchi Smiles Synonyms: avapritinib BLU-285 ayvakit	Avapritinib is a tyrosine kinase inhibitor that targets PDGFRA and PDGFRA D842 mutants as well as multiple KIT exon 11, 11/17 and 17 mutants with half maximal inhibitory concentrations (IC50s) less than 25 nM. Certain mutations in PDGFRA and KIT can result in the autophosphorylation and constitutive activation of these receptors which can contribute to tumor cell proliferation. Other potential targets for avapritinib include wild type KIT, PDGFRB, and CSFR1. Molecular weight: 498.57 Formula: C26H27FN10 CLOGP: 2.47 LIPINSKI: 0 HAC: 10 HDO: 1 TPSA: 106.29 ALOGS: -4.22 ROTB: 5 Status: ONP Legend: OFP - off patent OFM - off market ONP - on patent

Molecule

Description

Molfile Inchi Smiles

Synonyms:

avapritinib
BLU-285
ayvakit

Avapritinib is a tyrosine kinase inhibitor that targets PDGFRA and PDGFRA D842 mutants as well as multiple KIT exon 11, 11/17 and 17 mutants with half maximal inhibitory concentrations (IC50s) less than 25 nM. Certain mutations in PDGFRA and KIT can result in the autophosphorylation and constitutive activation of these receptors which can contribute to tumor cell proliferation. Other potential targets for avapritinib include wild type KIT, PDGFRB, and CSFR1.

Molecular weight: 498.57
Formula: C26H27FN10
CLOGP: 2.47
LIPINSKI: 0
HAC: 10
HDO: 1
TPSA: 106.29
ALOGS: -4.22
ROTB: 5

Status: ONP

Legend:
OFP - off patent
OFM - off market
ONP - on patent

Drug dosage:

Dose	Unit	Route
0.30	g	O

ADMET properties:

Property	Value	Reference
S (Water solubility)	0.00 mg/mL	Bocci G, Oprea TI, Benet LZ
BDDCS (Biopharmaceutical Drug Disposition Classification System)	2	Bocci G, Oprea TI, Benet LZ

Approvals:

Date	Agency	Company	Orphan
Sept. 24, 2020	EMA	Blueprint Medicines (Netherlands) B.V.
Jan. 9, 2020	FDA	BLUEPRINT MEDICINES

FDA Adverse Event Reporting System (Female)

MedDRA adverse event term	Likelihood ratio	Likelihood ratio threshold	Patients taking drug having adverse event	Patients taking drug not having adverse event	Patients not taking drug having adverse event	Patients not taking drug not having adverse event
Disease progression	69.05	45.18	25	295	122733	63365969

FDA Adverse Event Reporting System (Male)

MedDRA adverse event term	Likelihood ratio	Likelihood ratio threshold	Patients taking drug having adverse event	Patients taking drug not having adverse event	Patients not taking drug having adverse event	Patients not taking drug not having adverse event
Off label use	94.50	60.17	53	264	419471	34537143

FDA Adverse Event Reporting System (Geriatric)

MedDRA adverse event term	Likelihood ratio	Likelihood ratio threshold	Patients taking drug having adverse event	Patients taking drug not having adverse event	Patients not taking drug having adverse event	Patients not taking drug not having adverse event
Face oedema	53.33	46.35	15	444	28121	79715808

FDA Adverse Event Reporting System (Pediatric)

None

Pharmacologic Action:

Source	Code	Description
ATC	L01EX18	ANTINEOPLASTIC AND IMMUNOMODULATING AGENTS ANTINEOPLASTIC AGENTS PROTEIN KINASE INHIBITORS Other protein kinase inhibitors
FDA MoA	N0000020001	Tyrosine Kinase Inhibitors
FDA EPC	N0000175605	Kinase Inhibitor
FDA MoA	N0000185503	P-Glycoprotein Inhibitors
FDA MoA	N0000185504	Cytochrome P450 2C9 Inhibitors
FDA MoA	N0000190113	Breast Cancer Resistance Protein Inhibitors
FDA MoA	N0000191423	Multidrug and Toxin Extrusion Transporter 1 Inhibitors
FDA MoA	N0000193845	Bile Salt Export Pump Inhibitors
FDA MoA	N0000193932	Multidrug and Toxin Extrusion Transporter 2 K Inhibitors

Drug Use | Suggest Off label Use Form| |View source of the data|

Disease	Relation	SNOMED_ID	DOID
Systemic mast cell disease	indication	397016004	DOID:349
Gastrointestinal stromal tumor	indication	420120006	DOID:9253

🐶 Veterinary Drug Use

None

🐶 Veterinary products

None

Acid dissociation constants calculated using MoKa v3.0.0

Dissociation level	Dissociation constant	Type (acidic/basic)
pKa1	6.82	Basic
pKa2	6.22	Basic
pKa3	1.64	Basic
pKa4	1.05	Basic

Orange Book patent data (new drug applications)

Formulation strength	Trade name	Applicant	Application number	Approval date	Type	Dose form	Route	Patent number	Patent expiration date	Patent use
100MG	AYVAKIT	BLUEPRINT MEDICINES	N212608	Jan. 9, 2020	RX	TABLET	ORAL	9200002	Oct. 15, 2034	TREATMENT OF ADVANCED SYSTEMIC MASTOCYTOSIS, INCLUDING PATIENTS WITH AGGRESSIVE SYSTEMIC MASTOCYTOSIS (ASM), SYSTEMIC MASTOCYTOSIS WITH AN ASSOCIATED HEMATOLOGICAL NEOPLASM (SM-AHN), AND MAST CELL LEUKEMIA (MCL)
100MG	AYVAKIT	BLUEPRINT MEDICINES	N212608	Jan. 9, 2020	RX	TABLET	ORAL	9200002	Oct. 15, 2034	TREATMENT OF UNRESECTABLE OR METASTATIC GASTROINTESTINAL STROMAL TUMOR (GIST) HARBORING A PLATELET-DERIVED GROWTH FACTOR RECEPTOR ALPHA (PDGFRA) EXON 18 MUTATION
100MG	AYVAKIT	BLUEPRINT MEDICINES	N212608	Jan. 9, 2020	RX	TABLET	ORAL	9944651	Oct. 15, 2034	TREATMENT OF ADVANCED SYSTEMIC MASTOCYTOSIS, INCLUDING PATIENTS WITH AGGRESSIVE SYSTEMIC MASTOCYTOSIS (ASM), SYSTEMIC MASTOCYTOSIS WITH AN ASSOCIATED HEMATOLOGICAL NEOPLASM (SM-AHN), AND MAST CELL LEUKEMIA (MCL)
100MG	AYVAKIT	BLUEPRINT MEDICINES	N212608	Jan. 9, 2020	RX	TABLET	ORAL	9944651	Oct. 15, 2034	TREATMENT OF UNRESECTABLE OR METASTATIC GASTROINTESTINAL STROMAL TUMOR (GIST) HARBORING A PLATELET-DERIVED GROWTH FACTOR RECEPTOR ALPHA (PDGFRA) EXON 18 MUTATION
100MG	AYVAKIT	BLUEPRINT MEDICINES	N212608	Jan. 9, 2020	RX	TABLET	ORAL	9994575	Oct. 15, 2034	TREATMENT OF ADVANCED SYSTEMIC MASTOCYTOSIS, INCLUDING PATIENTS WITH AGGRESSIVE SYSTEMIC MASTOCYTOSIS (ASM), SYSTEMIC MASTOCYTOSIS WITH AN ASSOCIATED HEMATOLOGICAL NEOPLASM (SM-AHN), AND MAST CELL LEUKEMIA (MCL)
100MG	AYVAKIT	BLUEPRINT MEDICINES	N212608	Jan. 9, 2020	RX	TABLET	ORAL	9994575	Oct. 15, 2034	TREATMENT OF UNRESECTABLE OR METASTATIC GASTROINTESTINAL STROMAL TUMOR (GIST) HARBORING A PLATELET-DERIVED GROWTH FACTOR RECEPTOR ALPHA (PDGFRA) EXON 18 MUTATION
200MG	AYVAKIT	BLUEPRINT MEDICINES	N212608	Jan. 9, 2020	RX	TABLET	ORAL	9200002	Oct. 15, 2034	TREATMENT OF ADVANCED SYSTEMIC MASTOCYTOSIS, INCLUDING PATIENTS WITH AGGRESSIVE SYSTEMIC MASTOCYTOSIS (ASM), SYSTEMIC MASTOCYTOSIS WITH AN ASSOCIATED HEMATOLOGICAL NEOPLASM (SM-AHN), AND MAST CELL LEUKEMIA (MCL)
200MG	AYVAKIT	BLUEPRINT MEDICINES	N212608	Jan. 9, 2020	RX	TABLET	ORAL	9200002	Oct. 15, 2034	TREATMENT OF UNRESECTABLE OR METASTATIC GASTROINTESTINAL STROMAL TUMOR (GIST) HARBORING A PLATELET-DERIVED GROWTH FACTOR RECEPTOR ALPHA (PDGFRA) EXON 18 MUTATION
200MG	AYVAKIT	BLUEPRINT MEDICINES	N212608	Jan. 9, 2020	RX	TABLET	ORAL	9944651	Oct. 15, 2034	TREATMENT OF ADVANCED SYSTEMIC MASTOCYTOSIS, INCLUDING PATIENTS WITH AGGRESSIVE SYSTEMIC MASTOCYTOSIS (ASM), SYSTEMIC MASTOCYTOSIS WITH AN ASSOCIATED HEMATOLOGICAL NEOPLASM (SM-AHN), AND MAST CELL LEUKEMIA (MCL)
200MG	AYVAKIT	BLUEPRINT MEDICINES	N212608	Jan. 9, 2020	RX	TABLET	ORAL	9944651	Oct. 15, 2034	TREATMENT OF UNRESECTABLE OR METASTATIC GASTROINTESTINAL STROMAL TUMOR (GIST) HARBORING A PLATELET-DERIVED GROWTH FACTOR RECEPTOR ALPHA (PDGFRA) EXON 18 MUTATION
200MG	AYVAKIT	BLUEPRINT MEDICINES	N212608	Jan. 9, 2020	RX	TABLET	ORAL	9994575	Oct. 15, 2034	TREATMENT OF ADVANCED SYSTEMIC MASTOCYTOSIS, INCLUDING PATIENTS WITH AGGRESSIVE SYSTEMIC MASTOCYTOSIS (ASM), SYSTEMIC MASTOCYTOSIS WITH AN ASSOCIATED HEMATOLOGICAL NEOPLASM (SM-AHN), AND MAST CELL LEUKEMIA (MCL)
200MG	AYVAKIT	BLUEPRINT MEDICINES	N212608	Jan. 9, 2020	RX	TABLET	ORAL	9994575	Oct. 15, 2034	TREATMENT OF UNRESECTABLE OR METASTATIC GASTROINTESTINAL STROMAL TUMOR (GIST) HARBORING A PLATELET-DERIVED GROWTH FACTOR RECEPTOR ALPHA (PDGFRA) EXON 18 MUTATION
25MG	AYVAKIT	BLUEPRINT MEDICINES	N212608	June 16, 2021	RX	TABLET	ORAL	9200002	Oct. 15, 2034	TREATMENT OF ADVANCED SYSTEMIC MASTOCYTOSIS, INCLUDING PATIENTS WITH AGGRESSIVE SYSTEMIC MASTOCYTOSIS (ASM), SYSTEMIC MASTOCYTOSIS WITH AN ASSOCIATED HEMATOLOGICAL NEOPLASM (SM-AHN), AND MAST CELL LEUKEMIA (MCL)
25MG	AYVAKIT	BLUEPRINT MEDICINES	N212608	June 16, 2021	RX	TABLET	ORAL	9200002	Oct. 15, 2034	TREATMENT OF INDOLENT SYSTEMIC MASTOCYTOSIS (ISM)
25MG	AYVAKIT	BLUEPRINT MEDICINES	N212608	June 16, 2021	RX	TABLET	ORAL	9944651	Oct. 15, 2034	TREATMENT OF ADVANCED SYSTEMIC MASTOCYTOSIS, INCLUDING PATIENTS WITH AGGRESSIVE SYSTEMIC MASTOCYTOSIS (ASM), SYSTEMIC MASTOCYTOSIS WITH AN ASSOCIATED HEMATOLOGICAL NEOPLASM (SM-AHN), AND MAST CELL LEUKEMIA (MCL)
25MG	AYVAKIT	BLUEPRINT MEDICINES	N212608	June 16, 2021	RX	TABLET	ORAL	9944651	Oct. 15, 2034	TREATMENT OF INDOLENT SYSTEMIC MASTOCYTOSIS (ISM)
25MG	AYVAKIT	BLUEPRINT MEDICINES	N212608	June 16, 2021	RX	TABLET	ORAL	9994575	Oct. 15, 2034	TREATMENT OF ADVANCED SYSTEMIC MASTOCYTOSIS, INCLUDING PATIENTS WITH AGGRESSIVE SYSTEMIC MASTOCYTOSIS (ASM), SYSTEMIC MASTOCYTOSIS WITH AN ASSOCIATED HEMATOLOGICAL NEOPLASM (SM-AHN), AND MAST CELL LEUKEMIA (MCL)
25MG	AYVAKIT	BLUEPRINT MEDICINES	N212608	June 16, 2021	RX	TABLET	ORAL	9994575	Oct. 15, 2034	TREATMENT OF INDOLENT SYSTEMIC MASTOCYTOSIS (ISM)
300MG	AYVAKIT	BLUEPRINT MEDICINES	N212608	Jan. 9, 2020	RX	TABLET	ORAL	9200002	Oct. 15, 2034	TREATMENT OF UNRESECTABLE OR METASTATIC GASTROINTESTINAL STROMAL TUMOR (GIST) HARBORING A PLATELET-DERIVED GROWTH FACTOR RECEPTOR ALPHA (PDGFRA) EXON 18 MUTATION
300MG	AYVAKIT	BLUEPRINT MEDICINES	N212608	Jan. 9, 2020	RX	TABLET	ORAL	9944651	Oct. 15, 2034	TREATMENT OF UNRESECTABLE OR METASTATIC GASTROINTESTINAL STROMAL TUMOR (GIST) HARBORING A PLATELET-DERIVED GROWTH FACTOR RECEPTOR ALPHA (PDGFRA) EXON 18 MUTATION
300MG	AYVAKIT	BLUEPRINT MEDICINES	N212608	Jan. 9, 2020	RX	TABLET	ORAL	9994575	Oct. 15, 2034	TREATMENT OF UNRESECTABLE OR METASTATIC GASTROINTESTINAL STROMAL TUMOR (GIST) HARBORING A PLATELET-DERIVED GROWTH FACTOR RECEPTOR ALPHA (PDGFRA) EXON 18 MUTATION
50MG	AYVAKIT	BLUEPRINT MEDICINES	N212608	June 16, 2021	RX	TABLET	ORAL	9200002	Oct. 15, 2034	TREATMENT OF ADVANCED SYSTEMIC MASTOCYTOSIS, INCLUDING PATIENTS WITH AGGRESSIVE SYSTEMIC MASTOCYTOSIS (ASM), SYSTEMIC MASTOCYTOSIS WITH AN ASSOCIATED HEMATOLOGICAL NEOPLASM (SM-AHN), AND MAST CELL LEUKEMIA (MCL)
50MG	AYVAKIT	BLUEPRINT MEDICINES	N212608	June 16, 2021	RX	TABLET	ORAL	9944651	Oct. 15, 2034	TREATMENT OF ADVANCED SYSTEMIC MASTOCYTOSIS, INCLUDING PATIENTS WITH AGGRESSIVE SYSTEMIC MASTOCYTOSIS (ASM), SYSTEMIC MASTOCYTOSIS WITH AN ASSOCIATED HEMATOLOGICAL NEOPLASM (SM-AHN), AND MAST CELL LEUKEMIA (MCL)
50MG	AYVAKIT	BLUEPRINT MEDICINES	N212608	June 16, 2021	RX	TABLET	ORAL	9994575	Oct. 15, 2034	TREATMENT OF ADVANCED SYSTEMIC MASTOCYTOSIS, INCLUDING PATIENTS WITH AGGRESSIVE SYSTEMIC MASTOCYTOSIS (ASM), SYSTEMIC MASTOCYTOSIS WITH AN ASSOCIATED HEMATOLOGICAL NEOPLASM (SM-AHN), AND MAST CELL LEUKEMIA (MCL)

Orange Book exclusivity data (new drug applications)

Formulation strength	Trade name	Applicant	Application number	Approval date	Type	Dose form	Route	Exclusivity date	Description
100MG	AYVAKIT	BLUEPRINT MEDICINES	N212608	Jan. 9, 2020	RX	TABLET	ORAL	June 16, 2024	TREATMENT OF ADULT PATIENTS WITH ADVANCED SYSTEMIC MASTOCYTOSIS (ADVSM), INCLUDING PATIENTS WITH AGGRESSIVE SYSTEMIC MASTOCYTOSIS (ASM) AND SYSTEMIC MASTOCYTOSIS WITH AN ASSOCIATED HEMATOLOGICAL NEOPLASM (SM-AHN)
100MG	AYVAKIT	BLUEPRINT MEDICINES	N212608	Jan. 9, 2020	RX	TABLET	ORAL	June 16, 2024	TREATMENT OF ADULT PATIENTS WITH MAST CELL LEUKEMIA (MCL)
200MG	AYVAKIT	BLUEPRINT MEDICINES	N212608	Jan. 9, 2020	RX	TABLET	ORAL	June 16, 2024	TREATMENT OF ADULT PATIENTS WITH ADVANCED SYSTEMIC MASTOCYTOSIS (ADVSM), INCLUDING PATIENTS WITH AGGRESSIVE SYSTEMIC MASTOCYTOSIS (ASM) AND SYSTEMIC MASTOCYTOSIS WITH AN ASSOCIATED HEMATOLOGICAL NEOPLASM (SM-AHN)
200MG	AYVAKIT	BLUEPRINT MEDICINES	N212608	Jan. 9, 2020	RX	TABLET	ORAL	June 16, 2024	TREATMENT OF ADULT PATIENTS WITH MAST CELL LEUKEMIA (MCL)
25MG	AYVAKIT	BLUEPRINT MEDICINES	N212608	June 16, 2021	RX	TABLET	ORAL	June 16, 2024	TREATMENT OF ADULT PATIENTS WITH ADVANCED SYSTEMIC MASTOCYTOSIS (ADVSM), INCLUDING PATIENTS WITH AGGRESSIVE SYSTEMIC MASTOCYTOSIS (ASM) AND SYSTEMIC MASTOCYTOSIS WITH AN ASSOCIATED HEMATOLOGICAL NEOPLASM (SM-AHN)
25MG	AYVAKIT	BLUEPRINT MEDICINES	N212608	June 16, 2021	RX	TABLET	ORAL	June 16, 2024	TREATMENT OF ADULT PATIENTS WITH MAST CELL LEUKEMIA (MCL)
50MG	AYVAKIT	BLUEPRINT MEDICINES	N212608	June 16, 2021	RX	TABLET	ORAL	June 16, 2024	TREATMENT OF ADULT PATIENTS WITH ADVANCED SYSTEMIC MASTOCYTOSIS (ADVSM), INCLUDING PATIENTS WITH AGGRESSIVE SYSTEMIC MASTOCYTOSIS (ASM) AND SYSTEMIC MASTOCYTOSIS WITH AN ASSOCIATED HEMATOLOGICAL NEOPLASM (SM-AHN)
50MG	AYVAKIT	BLUEPRINT MEDICINES	N212608	June 16, 2021	RX	TABLET	ORAL	June 16, 2024	TREATMENT OF ADULT PATIENTS WITH MAST CELL LEUKEMIA (MCL)
100MG	AYVAKIT	BLUEPRINT MEDICINES	N212608	Jan. 9, 2020	RX	TABLET	ORAL	Jan. 9, 2025	NEW CHEMICAL ENTITY
200MG	AYVAKIT	BLUEPRINT MEDICINES	N212608	Jan. 9, 2020	RX	TABLET	ORAL	Jan. 9, 2025	NEW CHEMICAL ENTITY
25MG	AYVAKIT	BLUEPRINT MEDICINES	N212608	June 16, 2021	RX	TABLET	ORAL	Jan. 9, 2025	NEW CHEMICAL ENTITY
300MG	AYVAKIT	BLUEPRINT MEDICINES	N212608	Jan. 9, 2020	RX	TABLET	ORAL	Jan. 9, 2025	NEW CHEMICAL ENTITY
50MG	AYVAKIT	BLUEPRINT MEDICINES	N212608	June 16, 2021	RX	TABLET	ORAL	Jan. 9, 2025	NEW CHEMICAL ENTITY
25MG	AYVAKIT	BLUEPRINT MEDICINES	N212608	June 16, 2021	RX	TABLET	ORAL	May 22, 2026	TREATMENT OF ADULT PATIENTS WITH INDOLENT SYSTEMIC MASTOCYTOSIS (ISM)
100MG	AYVAKIT	BLUEPRINT MEDICINES	N212608	Jan. 9, 2020	RX	TABLET	ORAL	Jan. 9, 2027	INDICATED FOR THE TREATMENT OF ADULTS WITH UNRESECTABLE OR METASTATIC GIST HARBORING A PLATELET-DERIVED GROWTH FACTOR RECEPTOR ALPHA (PDGFRA) EXON 18 MUTATION, INCLUDING PDGFRA D842V MUTATIONS
200MG	AYVAKIT	BLUEPRINT MEDICINES	N212608	Jan. 9, 2020	RX	TABLET	ORAL	Jan. 9, 2027	INDICATED FOR THE TREATMENT OF ADULTS WITH UNRESECTABLE OR METASTATIC GIST HARBORING A PLATELET-DERIVED GROWTH FACTOR RECEPTOR ALPHA (PDGFRA) EXON 18 MUTATION, INCLUDING PDGFRA D842V MUTATIONS
25MG	AYVAKIT	BLUEPRINT MEDICINES	N212608	June 16, 2021	RX	TABLET	ORAL	Jan. 9, 2027	INDICATED FOR THE TREATMENT OF ADULTS WITH UNRESECTABLE OR METASTATIC GIST HARBORING A PLATELET-DERIVED GROWTH FACTOR RECEPTOR ALPHA (PDGFRA) EXON 18 MUTATION, INCLUDING PDGFRA D842V MUTATIONS
300MG	AYVAKIT	BLUEPRINT MEDICINES	N212608	Jan. 9, 2020	RX	TABLET	ORAL	Jan. 9, 2027	INDICATED FOR THE TREATMENT OF ADULTS WITH UNRESECTABLE OR METASTATIC GIST HARBORING A PLATELET-DERIVED GROWTH FACTOR RECEPTOR ALPHA (PDGFRA) EXON 18 MUTATION, INCLUDING PDGFRA D842V MUTATIONS
50MG	AYVAKIT	BLUEPRINT MEDICINES	N212608	June 16, 2021	RX	TABLET	ORAL	Jan. 9, 2027	INDICATED FOR THE TREATMENT OF ADULTS WITH UNRESECTABLE OR METASTATIC GIST HARBORING A PLATELET-DERIVED GROWTH FACTOR RECEPTOR ALPHA (PDGFRA) EXON 18 MUTATION, INCLUDING PDGFRA D842V MUTATIONS
100MG	AYVAKIT	BLUEPRINT MEDICINES	N212608	Jan. 9, 2020	RX	TABLET	ORAL	June 16, 2028	FOR THE TREATMENT OF ADULT PATIENTS WITH ADVANCED SYSTEMIC MASTOCYTOSIS (ADVSM). ADVSM INCLUDES PATIENTS WITH AGGRESSIVE SYSTEMIC MASTOCYTOSIS (ASM), SYSTEMIC MASTOCYTOSIS WITH AN ASSOCIATED HEMATOLOGICAL NEOPLASM (SM-AHN), AND MAST CELL LEUKEMIA (MCL)
200MG	AYVAKIT	BLUEPRINT MEDICINES	N212608	Jan. 9, 2020	RX	TABLET	ORAL	June 16, 2028	FOR THE TREATMENT OF ADULT PATIENTS WITH ADVANCED SYSTEMIC MASTOCYTOSIS (ADVSM). ADVSM INCLUDES PATIENTS WITH AGGRESSIVE SYSTEMIC MASTOCYTOSIS (ASM), SYSTEMIC MASTOCYTOSIS WITH AN ASSOCIATED HEMATOLOGICAL NEOPLASM (SM-AHN), AND MAST CELL LEUKEMIA (MCL)
25MG	AYVAKIT	BLUEPRINT MEDICINES	N212608	June 16, 2021	RX	TABLET	ORAL	June 16, 2028	FOR THE TREATMENT OF ADULT PATIENTS WITH ADVANCED SYSTEMIC MASTOCYTOSIS (ADVSM). ADVSM INCLUDES PATIENTS WITH AGGRESSIVE SYSTEMIC MASTOCYTOSIS (ASM), SYSTEMIC MASTOCYTOSIS WITH AN ASSOCIATED HEMATOLOGICAL NEOPLASM (SM-AHN), AND MAST CELL LEUKEMIA (MCL)
300MG	AYVAKIT	BLUEPRINT MEDICINES	N212608	Jan. 9, 2020	RX	TABLET	ORAL	June 16, 2028	FOR THE TREATMENT OF ADULT PATIENTS WITH ADVANCED SYSTEMIC MASTOCYTOSIS (ADVSM). ADVSM INCLUDES PATIENTS WITH AGGRESSIVE SYSTEMIC MASTOCYTOSIS (ASM), SYSTEMIC MASTOCYTOSIS WITH AN ASSOCIATED HEMATOLOGICAL NEOPLASM (SM-AHN), AND MAST CELL LEUKEMIA (MCL)
50MG	AYVAKIT	BLUEPRINT MEDICINES	N212608	June 16, 2021	RX	TABLET	ORAL	June 16, 2028	FOR THE TREATMENT OF ADULT PATIENTS WITH ADVANCED SYSTEMIC MASTOCYTOSIS (ADVSM). ADVSM INCLUDES PATIENTS WITH AGGRESSIVE SYSTEMIC MASTOCYTOSIS (ASM), SYSTEMIC MASTOCYTOSIS WITH AN ASSOCIATED HEMATOLOGICAL NEOPLASM (SM-AHN), AND MAST CELL LEUKEMIA (MCL)
100MG	AYVAKIT	BLUEPRINT MEDICINES	N212608	Jan. 9, 2020	RX	TABLET	ORAL	May 22, 2030	TREATMENT OF ADULT PATIENTS WITH INDOLENT SYSTEMIC MASTOCYTOSIS (ISM)
200MG	AYVAKIT	BLUEPRINT MEDICINES	N212608	Jan. 9, 2020	RX	TABLET	ORAL	May 22, 2030	TREATMENT OF ADULT PATIENTS WITH INDOLENT SYSTEMIC MASTOCYTOSIS (ISM)
25MG	AYVAKIT	BLUEPRINT MEDICINES	N212608	June 16, 2021	RX	TABLET	ORAL	May 22, 2030	TREATMENT OF ADULT PATIENTS WITH INDOLENT SYSTEMIC MASTOCYTOSIS (ISM)
300MG	AYVAKIT	BLUEPRINT MEDICINES	N212608	Jan. 9, 2020	RX	TABLET	ORAL	May 22, 2030	TREATMENT OF ADULT PATIENTS WITH INDOLENT SYSTEMIC MASTOCYTOSIS (ISM)
50MG	AYVAKIT	BLUEPRINT MEDICINES	N212608	June 16, 2021	RX	TABLET	ORAL	May 22, 2030	TREATMENT OF ADULT PATIENTS WITH INDOLENT SYSTEMIC MASTOCYTOSIS (ISM)

Bioactivity Summary:

Target	Class	Pharos	UniProt	Action	Type	Activity value (-log[M])	Bioact source	MoA source
Mast/stem cell growth factor receptor Kit	Kinase	P10721	KIT_HUMAN	INHIBITOR	IC50	9.57	SCIENTIFIC LITERATURE	DRUG LABEL
Platelet-derived growth factor receptor alpha	Kinase	P16234	PGFRA_HUMAN	INHIBITOR	IC50	9.62	SCIENTIFIC LITERATURE	DRUG LABEL
ATP-binding cassette sub-family G member 2	Transporter	Q9UNQ0	ABCG2_HUMAN		IC50	8.82	CHEMBL
Mast/stem cell growth factor receptor Kit	Kinase		KIT_MOUSE	INHIBITOR	IC50	7.66	SCIENTIFIC LITERATURE

External reference:

ID	Source
513P80B4YJ	UNII
C4743552	UMLSCUI
CHEMBL4204794	ChEMBL_ID
118023034	PUBCHEM_CID
DB15233	DRUGBANK_ID
10613	INN_ID
D11279	KEGG_DRUG
10368	IUPHAR_LIGAND_ID
018251	NDDF
838409003	SNOMEDCT_US
838462005	SNOMEDCT_US
4039082	VANDF
2272107	RXNORM
329235	MMSL
37922	MMSL
d09480	MMSL
C000707147	MESH_SUPPLEMENTAL_RECORD_UI

Pharmaceutical products:

Product	Category	Ingredients	NDC	Form	Quantity	Route	Marketing	Label
Ayvakit	HUMAN PRESCRIPTION DRUG LABEL	1	72064-110	TABLET, FILM COATED	100 mg	ORAL	NDA	32 sections
Ayvakit	HUMAN PRESCRIPTION DRUG LABEL	1	72064-110	TABLET, FILM COATED	100 mg	ORAL	NDA	32 sections
Ayvakit	HUMAN PRESCRIPTION DRUG LABEL	1	72064-120	TABLET, FILM COATED	200 mg	ORAL	NDA	32 sections
Ayvakit	HUMAN PRESCRIPTION DRUG LABEL	1	72064-120	TABLET, FILM COATED	200 mg	ORAL	NDA	32 sections
Ayvakit	HUMAN PRESCRIPTION DRUG LABEL	1	72064-125	TABLET, FILM COATED	25 mg	ORAL	NDA	32 sections
Ayvakit	HUMAN PRESCRIPTION DRUG LABEL	1	72064-125	TABLET, FILM COATED	25 mg	ORAL	NDA	32 sections
Ayvakit	HUMAN PRESCRIPTION DRUG LABEL	1	72064-130	TABLET, FILM COATED	300 mg	ORAL	NDA	32 sections
Ayvakit	HUMAN PRESCRIPTION DRUG LABEL	1	72064-130	TABLET, FILM COATED	300 mg	ORAL	NDA	32 sections
Ayvakit	HUMAN PRESCRIPTION DRUG LABEL	1	72064-150	TABLET, FILM COATED	50 mg	ORAL	NDA	32 sections
Ayvakit	HUMAN PRESCRIPTION DRUG LABEL	1	72064-150	TABLET, FILM COATED	50 mg	ORAL	NDA	32 sections