mitapivat 🐶 Veterinary Use | Indications/Contra | FAERs-F | FAERs-M | Orange Bk | PK/Tox | Related Drugs | BioActivity |

Stem definitionDrug idCAS RN
pyruvate kinase activators 5506 2151847-10-6

Description:

MoleculeDescription

Molfile Inchi Smiles

Synonyms:

  • mitapivat
  • mitapivat sulfate
  • pyrukynd
  • AG-348 sulfate hydrate
  • AG-348
Mitapivat is a pyruvate kinase activator that acts by allosterically binding to the pyruvate kinase tetramer and increasing pyruvate kinase (PK) activity. The red blood cell (RBC) form of pyruvate kinase (PK-R) is mutated in PK deficiency, which leads to reduced adenosine triphosphate(ATP), shortened RBC lifespan, and chronic hemolysis.
  • Molecular weight: 450.56
  • Formula: C24H26N4O3S
  • CLOGP: 3.44
  • LIPINSKI: 0
  • HAC: 7
  • HDO: 1
  • TPSA: 82.61
  • ALOGS: -4.14
  • ROTB: 5
  • Status: ONP
    • Legend:
    OFP - off patent
    OFM - off market
    ONP - on patent

Drug dosage:

DoseUnitRoute
0.10 g O

ADMET properties:

None

Predicted pharmacokinetic/toxicological properties (PK-AZ):

PropertyDescriptionValueUnitConfidenceSimilarity
kinase-safety-class ACVR2A,ACVR2B,AURKA,AXL,BRAF,BTK,CAMK2B,CAMK2D,CDK5,CDK9,DDR1,DYRK1B,EGFR,EIF2AK3,EPHB4,ERBB2,FLT3,GRK2,ITK,JAK2,MAP2K6,MAP3K21,MAP3K7,MAPK10,MAPK7,MAPK8,MAPK9,MAPKAPK2,MARK4,MET,MTOR,NTRK2,PDK1,PDK2,PDK4,PDPK1,PIK3CB,PIK3CD,PRKCD,PRKDC,SIK2,SIK3,STK33,SYK,TEK,TTK,ZAP70 47
kinase-selectivity-class AXL,ERBB2,GRK2,IKBKB,MAPK7,MAPK8,MAPKAPK2,PDK1,PDK4,SIK2,STK33,TEK,ULK1 13
pfas-class PFAS structural alert (1 = True, 0 = False) 0

Approvals:

DateAgencyCompanyOrphan
Feb. 17, 2022 FDA AGIOS PHARMACEUTICALS INC
Nov. 9, 2022 EMA AGIOS NETHERLANDS B.V.

FDA Adverse Event Reporting System (Female)

None

FDA Adverse Event Reporting System (Male)

MedDRA adverse event termLikelihood ratioLikelihood ratio thresholdPatients taking drug having adverse eventPatients taking drug not having adverse eventPatients not taking drug having adverse eventPatients not taking drug not having adverse event
Haemoglobin decreased 54.36 44.33 17 75 132454 42488789

FDA Adverse Event Reporting System (Geriatric)

None

FDA Adverse Event Reporting System (Pediatric)

None

Pharmacologic Action:

SourceCodeDescription
ATC B06AX04 BLOOD AND BLOOD FORMING ORGANS
OTHER HEMATOLOGICAL AGENTS
OTHER HEMATOLOGICAL AGENTS
Other hematological agents
MeSH PA D020536 Enzyme Activators
FDA MoA N0000185503 P-Glycoprotein Inhibitors
FDA MoA N0000185507 Cytochrome P450 2C9 Inducers
FDA MoA N0000185607 Cytochrome P450 2C19 Inducers
FDA MoA N0000187063 Cytochrome P450 2C8 Inducers
FDA MoA N0000187064 Cytochrome P450 2B6 Inducers
FDA MoA N0000190118 Cytochrome P450 3A Inducers
FDA EPC N0000194008 Pyruvate Kinase Activator
FDA MoA N0000194009 Pyruvate Kinase Activators
FDA MoA N0000194036 UGT1A1 Inducers
CHEBI has role CHEBI:75835 anti-anaemic agent

Related Drugs by ATC class:

B06AX Other hematological agents 2 drugs
crizanlizumabvoxelotor

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

DiseaseRelationSNOMED_IDDOID
Pyruvate kinase (PK) deficiency indication 124331002




🐶 Veterinary Drug Use

None

🐶 Veterinary products

None

Acid dissociation constants calculated using MoKa v3.0.0

None

Orange Book patent data (new drug applications)

Formulation strengthTrade nameApplicantApplication numberApproval dateTypeDose formRoutePatent numberPatent expiration datePatent use
EQ 100MG BASE AQVESME AGIOS PHARMS INC N216196 Dec. 23, 2025 RX TABLET ORAL 10632114 May 3, 2032 METHOD OF USING A PYRUVATE KINASE ACTIVATOR FOR TREATING ANEMIA IN ADULTS WITH ALPHA OR BETA THALASSEMIA
EQ 100MG BASE AQVESME AGIOS PHARMS INC N216196 Dec. 23, 2025 RX TABLET ORAL 9682080 May 3, 2032 METHOD OF USING A PYRUVATE KINASE ACTIVATOR FOR THE TREATMENT OF ANEMIA IN ADULTS WITH ALPHA OR BETA THALASSEMIA
EQ 20MG BASE PYRUKYND AGIOS PHARMS INC N216196 Feb. 17, 2022 RX TABLET ORAL 10632114 May 3, 2032 METHOD FOR INCREASING THE LIFETIME OF RED BLOOD CELLS (RBCS) FOR THE TREATMENT OF HEMOLYTIC ANEMIA IN ADULTS WITH PYRUVATE KINASE (PK) DEFICIENCY
EQ 20MG BASE PYRUKYND AGIOS PHARMS INC N216196 Feb. 17, 2022 RX TABLET ORAL 9682080 May 3, 2032 METHOD OF USING A PYRUVATE KINASE ACTIVATOR FOR THE TREATMENT OF HEMOLYTIC ANEMIA IN ADULTS WITH PYRUVATE KINASE (PK) DEFICIENCY
EQ 20MG BASE PYRUKYND AGIOS PHARMS INC N216196 Feb. 17, 2022 RX TABLET ORAL 9980961 May 3, 2032 METHOD OF USING A PYRUVATE KINASE ACTIVATOR FOR THE TREATMENT OF HEMOLYTIC ANEMIA IN ADULTS WITH PYRUVATE KINASE (PK) DEFICIENCY
EQ 50MG BASE PYRUKYND AGIOS PHARMS INC N216196 Feb. 17, 2022 RX TABLET ORAL 10632114 May 3, 2032 METHOD FOR INCREASING THE LIFETIME OF RED BLOOD CELLS (RBCS) FOR THE TREATMENT OF HEMOLYTIC ANEMIA IN ADULTS WITH PYRUVATE KINASE (PK) DEFICIENCY
EQ 50MG BASE PYRUKYND AGIOS PHARMS INC N216196 Feb. 17, 2022 RX TABLET ORAL 9682080 May 3, 2032 METHOD OF USING A PYRUVATE KINASE ACTIVATOR FOR THE TREATMENT OF HEMOLYTIC ANEMIA IN ADULTS WITH PYRUVATE KINASE (PK) DEFICIENCY
EQ 50MG BASE PYRUKYND AGIOS PHARMS INC N216196 Feb. 17, 2022 RX TABLET ORAL 9980961 May 3, 2032 METHOD OF USING A PYRUVATE KINASE ACTIVATOR FOR THE TREATMENT OF HEMOLYTIC ANEMIA IN ADULTS WITH PYRUVATE KINASE (PK) DEFICIENCY
EQ 5MG BASE PYRUKYND AGIOS PHARMS INC N216196 Feb. 17, 2022 RX TABLET ORAL 10632114 May 3, 2032 METHOD FOR INCREASING THE LIFETIME OF RED BLOOD CELLS (RBCS) FOR THE TREATMENT OF HEMOLYTIC ANEMIA IN ADULTS WITH PYRUVATE KINASE (PK) DEFICIENCY
EQ 5MG BASE PYRUKYND AGIOS PHARMS INC N216196 Feb. 17, 2022 RX TABLET ORAL 9682080 May 3, 2032 METHOD OF USING A PYRUVATE KINASE ACTIVATOR FOR THE TREATMENT OF HEMOLYTIC ANEMIA IN ADULTS WITH PYRUVATE KINASE (PK) DEFICIENCY
EQ 5MG BASE PYRUKYND AGIOS PHARMS INC N216196 Feb. 17, 2022 RX TABLET ORAL 9980961 May 3, 2032 METHOD OF USING A PYRUVATE KINASE ACTIVATOR FOR THE TREATMENT OF HEMOLYTIC ANEMIA IN ADULTS WITH PYRUVATE KINASE (PK) DEFICIENCY
EQ 100MG BASE AQVESME AGIOS PHARMS INC N216196 Dec. 23, 2025 RX TABLET ORAL 9193701 Oct. 26, 2032 METHOD OF USING A PYRUVATE KINASE ACTIVATOR FOR INCREASING RED BLOOD CELL LONGEVITY IN ADULTS WITH ALPHA OR BETA THALASSEMIA
EQ 20MG BASE PYRUKYND AGIOS PHARMS INC N216196 Feb. 17, 2022 RX TABLET ORAL 9193701 Oct. 26, 2032 METHOD OF USING A PYRUVATE KINASE ACTIVATOR FOR THE TREATMENT OF HEMOLYTIC ANEMIA IN ADULTS WITH PYRUVATE KINASE (PK) DEFICIENCY
EQ 50MG BASE PYRUKYND AGIOS PHARMS INC N216196 Feb. 17, 2022 RX TABLET ORAL 9193701 Oct. 26, 2032 METHOD OF USING A PYRUVATE KINASE ACTIVATOR FOR THE TREATMENT OF HEMOLYTIC ANEMIA IN ADULTS WITH PYRUVATE KINASE (PK) DEFICIENCY
EQ 5MG BASE PYRUKYND AGIOS PHARMS INC N216196 Feb. 17, 2022 RX TABLET ORAL 9193701 Oct. 26, 2032 METHOD OF USING A PYRUVATE KINASE ACTIVATOR FOR THE TREATMENT OF HEMOLYTIC ANEMIA IN ADULTS WITH PYRUVATE KINASE (PK) DEFICIENCY
EQ 20MG BASE PYRUKYND AGIOS PHARMS INC N216196 Feb. 17, 2022 RX TABLET ORAL 11793806 April 12, 2033 METHOD FOR INCREASING THE LIFETIME OF RED BLOOD CELLS (RBCS) FOR THE TREATMENT OF HEMOLYTIC ANEMIA IN ADULTS WITH PYRUVATE KINASE (PK) DEFICIENCY
EQ 50MG BASE PYRUKYND AGIOS PHARMS INC N216196 Feb. 17, 2022 RX TABLET ORAL 11793806 April 12, 2033 METHOD FOR INCREASING THE LIFETIME OF RED BLOOD CELLS (RBCS) FOR THE TREATMENT OF HEMOLYTIC ANEMIA IN ADULTS WITH PYRUVATE KINASE (PK) DEFICIENCY
EQ 5MG BASE PYRUKYND AGIOS PHARMS INC N216196 Feb. 17, 2022 RX TABLET ORAL 11793806 April 12, 2033 METHOD FOR INCREASING THE LIFETIME OF RED BLOOD CELLS (RBCS) FOR THE TREATMENT OF HEMOLYTIC ANEMIA IN ADULTS WITH PYRUVATE KINASE (PK) DEFICIENCY
EQ 20MG BASE PYRUKYND AGIOS PHARMS INC N216196 Feb. 17, 2022 RX TABLET ORAL 11234976 Oct. 11, 2038 METHOD OF USING A PYRUVATE KINASE ACTIVATOR FOR THE TREATMENT OF HEMOLYTIC ANEMIA IN ADULTS WITH PYRUVATE KINASE (PK) DEFICIENCY BY ADMINISTERING A DAILY DOSE OF 10MG TO 100MG
EQ 50MG BASE PYRUKYND AGIOS PHARMS INC N216196 Feb. 17, 2022 RX TABLET ORAL 11234976 Oct. 11, 2038 METHOD OF USING A PYRUVATE KINASE ACTIVATOR FOR THE TREATMENT OF HEMOLYTIC ANEMIA IN ADULTS WITH PYRUVATE KINASE (PK) DEFICIENCY BY ADMINISTERING A DAILY DOSE OF 10MG TO 100MG
EQ 5MG BASE PYRUKYND AGIOS PHARMS INC N216196 Feb. 17, 2022 RX TABLET ORAL 11234976 Oct. 11, 2038 METHOD OF USING A PYRUVATE KINASE ACTIVATOR FOR THE TREATMENT OF HEMOLYTIC ANEMIA IN ADULTS WITH PYRUVATE KINASE (PK) DEFICIENCY BY ADMINISTERING A DAILY DOSE OF 10MG TO 100MG
EQ 100MG BASE AQVESME AGIOS PHARMS INC N216196 Dec. 23, 2025 RX TABLET ORAL 11878049 July 31, 2041 METHOD FOR USING A PYRUVATE KINASE ACTIVATOR TO MITIGATE DRUG INTERACTIONS IN ADULTS WITH HEMOLYTIC ANEMIA THAT ARE TAKING MODERATE CYP3A INDUCERS
EQ 20MG BASE PYRUKYND AGIOS PHARMS INC N216196 Feb. 17, 2022 RX TABLET ORAL 11878049 July 31, 2041 METHOD FOR ADMINISTERING MITAPIVAT OR A SALT OF MITAPIVAT TO MITIGATE DRUG INTERACTIONS IN PATIENTS WITH HEMOLYTIC ANEMIA THAT ARE TAKING MODERATE CYP3A INDUCERS
EQ 50MG BASE PYRUKYND AGIOS PHARMS INC N216196 Feb. 17, 2022 RX TABLET ORAL 11878049 July 31, 2041 METHOD FOR ADMINISTERING MITAPIVAT OR A SALT OF MITAPIVAT TO MITIGATE DRUG INTERACTIONS IN PATIENTS WITH HEMOLYTIC ANEMIA THAT ARE TAKING MODERATE CYP3A INDUCERS
EQ 5MG BASE PYRUKYND AGIOS PHARMS INC N216196 Feb. 17, 2022 RX TABLET ORAL 11878049 July 31, 2041 METHOD FOR ADMINISTERING MITAPIVAT OR A SALT OF MITAPIVAT TO MITIGATE DRUG INTERACTIONS IN PATIENTS WITH HEMOLYTIC ANEMIA THAT ARE TAKING MODERATE CYP3A INDUCERS

Orange Book exclusivity data (new drug applications)

Formulation strengthTrade nameApplicantApplication numberApproval dateTypeDose formRouteExclusivity dateDescription
EQ 100MG BASE AQVESME AGIOS PHARMS INC N216196 Dec. 23, 2025 RX TABLET ORAL Feb. 17, 2027 NEW CHEMICAL ENTITY
EQ 20MG BASE PYRUKYND AGIOS PHARMS INC N216196 Feb. 17, 2022 RX TABLET ORAL Feb. 17, 2027 NEW CHEMICAL ENTITY
EQ 50MG BASE PYRUKYND AGIOS PHARMS INC N216196 Feb. 17, 2022 RX TABLET ORAL Feb. 17, 2027 NEW CHEMICAL ENTITY
EQ 5MG BASE PYRUKYND AGIOS PHARMS INC N216196 Feb. 17, 2022 RX TABLET ORAL Feb. 17, 2027 NEW CHEMICAL ENTITY
EQ 100MG BASE AQVESME AGIOS PHARMS INC N216196 Dec. 23, 2025 RX TABLET ORAL Dec. 23, 2028 NEW PRODUCT
EQ 20MG BASE PYRUKYND AGIOS PHARMS INC N216196 Feb. 17, 2022 RX TABLET ORAL Feb. 17, 2029 TREATMENT OF HEMOLYTIC ANEMIA IN ADULTS WITH PYRUVATE KINASE (PK) DEFICIENCY
EQ 50MG BASE PYRUKYND AGIOS PHARMS INC N216196 Feb. 17, 2022 RX TABLET ORAL Feb. 17, 2029 TREATMENT OF HEMOLYTIC ANEMIA IN ADULTS WITH PYRUVATE KINASE (PK) DEFICIENCY
EQ 5MG BASE PYRUKYND AGIOS PHARMS INC N216196 Feb. 17, 2022 RX TABLET ORAL Feb. 17, 2029 TREATMENT OF HEMOLYTIC ANEMIA IN ADULTS WITH PYRUVATE KINASE (PK) DEFICIENCY
EQ 100MG BASE AQVESME AGIOS PHARMS INC N216196 Dec. 23, 2025 RX TABLET ORAL Dec. 23, 2032 TREATMENT OF ANEMIA IN ADULTS WITH ALPHA-OR BETA-THALASSEMIA

Bioactivity Summary:

TargetClassPharosUniProtActionTypeActivity value
(-log[M])		Mechanism
action		Bioact sourceMoA source
Pyruvate kinase PKLR Enzyme ACTIVATOR AC50 7.89 DRUG LABEL DRUG LABEL
Pyruvate kinase PKM Enzyme ACTIVATOR AC50 7.42 DRUG LABEL

External reference:

IDSource
1260075-17-9 SECONDARY_CAS_RN
CHEBI:757622 CHEBI
WV2 PDB_CHEM_ID
CHEMBL4299940 ChEMBL_ID
C000634504 MESH_SUPPLEMENTAL_RECORD_UI
10473 IUPHAR_LIGAND_ID
DB16236 DRUGBANK_ID
42782611000001109 SNOMEDCT_US
44102311000001100 SNOMEDCT_US
4041222 VANDF
C5226639 UMLSCUI
CHEMBL4297223 ChEMBL_ID
D11408 KEGG_DRUG
10226 INN_ID
59634741 PUBCHEM_CID
357746 MMSL
40529 MMSL
40563 MMSL
d09864 MMSL
018958 NDDF
018959 NDDF
2WTV10SIKH UNII
2594468 RXNORM
2WTV10SIKH INXIGHT DRUGS

Pharmaceutical products:

ProductCategoryIngredientsNDCFormQuantityRouteMarketingLabel
PYRUKYND HUMAN PRESCRIPTION DRUG LABEL 1 71334-205 TABLET, FILM COATED 5 mg ORAL NDA 27 sections
PYRUKYND HUMAN PRESCRIPTION DRUG LABEL 1 71334-210 TABLET, FILM COATED 20 mg ORAL NDA 27 sections
PYRUKYND HUMAN PRESCRIPTION DRUG LABEL 1 71334-215 TABLET, FILM COATED 50 mg ORAL NDA 27 sections
PYRUKYND HUMAN PRESCRIPTION DRUG LABEL 1 71334-220 TABLET, FILM COATED 5 mg ORAL NDA 27 sections
AQVESME HUMAN PRESCRIPTION DRUG LABEL 1 71334-235 TABLET, FILM COATED 100 mg ORAL NDA 26 sections