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Chemistry

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Also known as: Blu-285, 1703793-34-3, Ayvakit, Avapritinib [inn], Blu285, (1s)-1-(4-fluorophenyl)-1-[2-[4-[6-(1-methylpyrazol-4-yl)pyrrolo[2,1-f][1,2,4]triazin-4-yl]piperazin-1-yl]pyrimidin-5-yl]ethanamine
Molecular Formula
C26H27FN10
Molecular Weight
498.6  g/mol
InChI Key
DWYRIWUZIJHQKQ-SANMLTNESA-N
FDA UNII
513P80B4YJ

Avapritinib
Avapritinib is an orally bioavailable inhibitor of specific mutated forms of platelet-derived growth factor receptor alpha (PDGFR alpha; PDGFRa) and mast/stem cell factor receptor c-Kit (SCFR), with potential antineoplastic activity. Upon oral administration, avapritinib specifically binds to and inhibits specific mutant forms of PDGFRa and c-Kit, including the PDGFRa D842V mutant and various KIT exon 17 mutants. This results in the inhibition of PDGFRa- and c-Kit-mediated signal transduction pathways and the inhibition of proliferation in tumor cells that express these PDGFRa and c-Kit mutants. PDGFRa and c-Kit, protein tyrosine kinases and tumor-associated antigens (TAAs), are mutated in various tumor cell types; they play key roles in the regulation of cellular proliferation.
Avapritinib is a Kinase Inhibitor. The mechanism of action of avapritinib is as a Tyrosine Kinase Inhibitor, and Cytochrome P450 2C9 Inhibitor, and P-Glycoprotein Inhibitor, and Breast Cancer Resistance Protein Inhibitor, and Multidrug and Toxin Extrusion Transporter 1 Inhibitor, and Multidrug and Toxin Extrusion Transporter 2 K Inhibitor, and Bile Salt Export Pump Inhibitor.
1 2D Structure

Avapritinib

2 Identification
2.1 Computed Descriptors
2.1.1 IUPAC Name
(1S)-1-(4-fluorophenyl)-1-[2-[4-[6-(1-methylpyrazol-4-yl)pyrrolo[2,1-f][1,2,4]triazin-4-yl]piperazin-1-yl]pyrimidin-5-yl]ethanamine
2.1.2 InChI
InChI=1S/C26H27FN10/c1-26(28,20-3-5-22(27)6-4-20)21-13-29-25(30-14-21)36-9-7-35(8-10-36)24-23-11-18(16-37(23)33-17-31-24)19-12-32-34(2)15-19/h3-6,11-17H,7-10,28H2,1-2H3/t26-/m0/s1
2.1.3 InChI Key
DWYRIWUZIJHQKQ-SANMLTNESA-N
2.1.4 Canonical SMILES
CC(C1=CC=C(C=C1)F)(C2=CN=C(N=C2)N3CCN(CC3)C4=NC=NN5C4=CC(=C5)C6=CN(N=C6)C)N
2.1.5 Isomeric SMILES
C[C@](C1=CC=C(C=C1)F)(C2=CN=C(N=C2)N3CCN(CC3)C4=NC=NN5C4=CC(=C5)C6=CN(N=C6)C)N
2.2 Other Identifiers
2.2.1 UNII
513P80B4YJ
2.3 Synonyms
2.3.1 MeSH Synonyms

1. (1s)-1-(4-fluorophenyl)-1-(2-(4-(6-(1-methyl-1h-pyrazol-4-yl)pyrrolo(2,1-f)(1,2,4)triazin-4-yl)-1-piperazinyl)-5-pyrimidinyl)ethanamine

2. 5-pyrimidinemethanamine, Alpha-(4-fluorophenyl)-alpha-methyl-2-(4-(6-(1-methyl-1h-pyrazol-4-yl)pyrrolo(2,1-f)(1,2,4)triazin-4-yl)-1-piperazinyl)-, (alphas)-

3. Ayvakit

4. Blu-285

2.3.2 Depositor-Supplied Synonyms

1. Blu-285

2. 1703793-34-3

3. Ayvakit

4. Avapritinib [inn]

5. Blu285

6. (1s)-1-(4-fluorophenyl)-1-[2-[4-[6-(1-methylpyrazol-4-yl)pyrrolo[2,1-f][1,2,4]triazin-4-yl]piperazin-1-yl]pyrimidin-5-yl]ethanamine

7. 513p80b4yj

8. C-366

9. 70c366

10. X720776

11. X-720776

12. Ayvakyt

13. Mfcd31544325

14. Ayvakit (tn)

15. Avapritinib [mi]

16. Avapritinib (usan/inn)

17. Avapritinib [usan]

18. Avapritinib (blu-285)

19. Blu-285 (avapritinib)

20. Avapritinib [who-dd]

21. Unii-513p80b4yj

22. Chembl4204794

23. Schembl16652297

24. Gtpl10368

25. Avapritinib [orange Book]

26. Blu 285

27. Bdbm469269

28. Dtxsid301027935

29. Amy16753

30. Ex-a1366

31. Us10807985, Compound 44

32. Nsc801082

33. S8553

34. Akos037648993

35. Ccg-269677

36. Cs-7577

37. Db15233

38. Nsc-801082

39. (s)-1-(4-fluorophenyl)-1-(2-(4-(6-(1-methyl-1h-pyrazol-4-yl)pyrrolo[2,1-f][1,2,4]triazin-4-yl)piperazin-1-yl)pyrimidin-5-yl)ethan-1-amine

40. Ac-31598

41. Bb166456

42. Bs-16206

43. Hy-101561

44. D11279

45. Q29213676

46. (1s)-1-(4-fluorophenyl)-1-(2-(4-(6-(1-methyl-1h-pyrazol-4-yl)pyrrolo(2,1-f)(1,2,4)triazin-4-yl)-1-piperazinyl)-5-pyrimidinyl)ethanamine

47. (1s)-1-(4-fluorophenyl)-1-(2-(4-(6-(1-methyl-1h-pyrazol-4-yl)pyrrolo(2,1-f)(1,2,4)triazin-4-yl)piperazin-1-yl)pyrimidin-5-yl)ethan-1-amine

48. (s)-1-(4-fluorophenyl)-1-(2-(4-(6-(1-methyl-1h-pyrazol-4-yl)pyrrolo(2,1-f)(1,2,4)triazin-4-yl)piperazin-yl)pyrimidin-5-yl)ethan-1-amine

49. (s)-1-(4-fluorophenyl)-1-(2-(4-(6-(1-methyl-1h-pyrazol-4-yl)pyrrolo[2,1-f][1,2,4]triazin-4-yl)piperazin-1-yl)pyrimidin-5-yl)ethanamine

50. 5-pyrimidinemethanamine, .alpha.-(4-fluorophenyl)-.alpha.-methyl-2-(4-(6-(1-methyl-1h-pyrazol-4-yl)pyrrolo(2,1-f)(1,2,4)triazin-4-yl)-1-piperazinyl)-, (.alpha.s)-

2.4 Create Date
2016-02-23
3 Chemical and Physical Properties
Molecular Weight 498.6 g/mol
Molecular Formula C26H27FN10
XLogP31.9
Hydrogen Bond Donor Count1
Hydrogen Bond Acceptor Count9
Rotatable Bond Count5
Exact Mass498.24041907 g/mol
Monoisotopic Mass498.24041907 g/mol
Topological Polar Surface Area106 Ų
Heavy Atom Count37
Formal Charge0
Complexity752
Isotope Atom Count0
Defined Atom Stereocenter Count1
Undefined Atom Stereocenter Count0
Defined Bond Stereocenter Count0
Undefined Bond Stereocenter Count0
Covalently Bonded Unit Count1
4 Drug and Medication Information
4.1 Drug Indication

Avapritinib is indicated for the treatment of unresectable, metastatic gastrointestinal stromal tumors with a platelet-derived growth factor receptor alpha exon 18 mutation.


Treatment of mastocytosis


Treatment of all conditions included in the category of malignant neoplasms (except haematopoietic and lymphoid tissue neoplasms)


Ayvakyt is indicated as monotherapy for the treatment of adult patients with unresectable or metastatic gastrointestinal stromal tumours (GIST) harbouring the platelet-derived growth factor receptor alpha (PDGFRA) D842V mutation.


5 Pharmacology and Biochemistry
5.1 Pharmacology

Avapritinib is a selective kinase inhibitor that negatively modulates the action of cell transporters to resensitize them to other chemotherapies. It has a long duration of action as it is given once daily. Patients should be counselled regarding the risk of intracranial hemorrhage, CNS effects, and embryo-fetal toxicity.


5.2 FDA Pharmacological Classification
5.2.1 Active Moiety
AVAPRITINIB
5.2.2 FDA UNII
513P80B4YJ
5.2.3 Pharmacological Classes
Breast Cancer Resistance Protein Inhibitors [MoA]; Cytochrome P450 2C9 Inhibitors [MoA]; Kinase Inhibitor [EPC]; Multidrug and Toxin Extrusion Transporter 1 Inhibitors [MoA]; Multidrug and Toxin Extrusion Transporter 2 K Inhibitors [MoA]; P-Glycoprotein Inhibitors [MoA]; Tyrosine Kinase Inhibitors [MoA]; Bile Salt Export Pump Inhibitors [MoA]
5.3 ATC Code

L01EX18


L - Antineoplastic and immunomodulating agents

L01 - Antineoplastic agents

L01E - Protein kinase inhibitors

L01EX - Other protein kinase inhibitors

L01EX18 - Avapritinib


5.4 Absorption, Distribution and Excretion

Absorption

A 300mg oral dose of avapritinib reaches a Cmax of 813ng/mL with a Tmax of 2.0-4.1h and an AUC of 15400h\*ng/mL.


Route of Elimination

Avapritinib is 70% eliminated in the feces with 11% as the unchanged drug and 18% eliminated in the urine with 0.23% as the unchanged drug.


Volume of Distribution

The mean apparent volume of distribution is 1200L.


Clearance

The mean apparent oral clearance of avapritinib is 19.5L/h.


5.5 Metabolism/Metabolites

Avapritinib is metabolized mainly by CYP3A4 and CYP2C9 in vitro. A 310mg oral dose is recovered as 49% unchanged drug, 35% hydroxy glucuronide metabolite, and 14% oxidatively deaminated metabolite.


5.6 Biological Half-Life

The half life of avapritinib is 32-57h.


5.7 Mechanism of Action

Avapritinib has a negative modulating effect on the transporters ABCB1 and ABCG2, which mediate the multidrug resistance phenotype of some cancers. This modulation may be due to interactions of avapritinib with the drug binding pocket of these transporters. Negative modulation of these transporters, resensitizes cancerous cells to treatment with chemotherapeutic agents like [paclitaxel].


API SUPPLIERS

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Egis Pharmaceuticals PLC

Hungary

USDMF, CEP/COS, JDMF, EU-WC, NDC, KDMF, VMF, Others

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TAPI Technology & API Services

Israel

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Virtual BoothTAPI, a leading global supplier of APIs, provides over 350 products and customized CDMO solutions for every stage of development.

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Beijing Sjar Technology Developmen...

China

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AAPS National Biotech
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Drugs in Development

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Details:

Ayvakyt (avapritinib) is a kinase inhibitor, works by potently and selectively target KIT D816V. It is approved by european commission for the treatment of indolent systemic mastocytosis (ISM).


Lead Product(s): Avapritinib

Therapeutic Area: Hematology Brand Name: Ayvakyt

Study Phase: Approved FDFProduct Type: Other Small Molecule

Sponsor: Inapplicable

Deal Size: Inapplicable Upfront Cash: Inapplicable

Deal Type: Inapplicable December 13, 2023

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01

India Med Expo
Not Confirmed
India Med Expo
Not Confirmed

Details : Ayvakyt (avapritinib) is a kinase inhibitor, works by potently and selectively target KIT D816V. It is approved by european commission for the treatment of indolent systemic mastocytosis (ISM).

Product Name : Ayvakyt

Product Type : Other Small Molecule

Upfront Cash : Inapplicable

December 13, 2023

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Details:

AYVAKYT® (avapritinib) is a kinase inhibitor approved by the European Commission for the treatment of adult patients with aggressive systemic mastocytosis (ASM), systemic mastocytosis with an associated hematological neoplasm (SM-AHN) or mast cell leukemia (MCL).


Lead Product(s): Avapritinib

Therapeutic Area: Hematology Brand Name: Ayvakit

Study Phase: Approved FDFProduct Type: Other Small Molecule

Sponsor: Inapplicable

Deal Size: Inapplicable Upfront Cash: Inapplicable

Deal Type: Inapplicable October 11, 2023

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02

India Med Expo
Not Confirmed
India Med Expo
Not Confirmed

Details : AYVAKYT® (avapritinib) is a kinase inhibitor approved by the European Commission for the treatment of adult patients with aggressive systemic mastocytosis (ASM), systemic mastocytosis with an associated hematological neoplasm (SM-AHN) or mast cell leuke...

Product Name : Ayvakit

Product Type : Other Small Molecule

Upfront Cash : Inapplicable

October 11, 2023

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Details:

Ayvakit (avapritinib) is a FDA approved kinase inhibitor for treatment adults with Indolent Systemic Mastocytosis, ASM, SM-AHN and MCL, also for adults with GIST harboring a PDGFRA exon 18 mutation, including PDGFRA D842V mutations.


Lead Product(s): Avapritinib

Therapeutic Area: Hematology Brand Name: Ayvakit

Study Phase: Approved FDFProduct Type: Other Small Molecule

Sponsor: Inapplicable

Deal Size: Inapplicable Upfront Cash: Inapplicable

Deal Type: Inapplicable May 22, 2023

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03

India Med Expo
Not Confirmed
India Med Expo
Not Confirmed

Details : Ayvakit (avapritinib) is a FDA approved kinase inhibitor for treatment adults with Indolent Systemic Mastocytosis, ASM, SM-AHN and MCL, also for adults with GIST harboring a PDGFRA exon 18 mutation, including PDGFRA D842V mutations.

Product Name : Ayvakit

Product Type : Other Small Molecule

Upfront Cash : Inapplicable

May 22, 2023

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Details:

Ayvakit (avapritinib) is a kinase inhibitor approved by the FDA for the treatment of adults with Advanced SM, ASM, SM-AHN and MCL, and adults with unresectable or metastatic GIST harboring a PDGFRA exon 18 mutation, including PDGFRA D842V mutations.


Lead Product(s): Avapritinib

Therapeutic Area: Hematology Brand Name: Ayvakit

Study Phase: Phase IIIProduct Type: Other Small Molecule

Sponsor: Inapplicable

Deal Size: Inapplicable Upfront Cash: Inapplicable

Deal Type: Inapplicable March 02, 2023

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04

India Med Expo
Not Confirmed
India Med Expo
Not Confirmed

Details : Ayvakit (avapritinib) is a kinase inhibitor approved by the FDA for the treatment of adults with Advanced SM, ASM, SM-AHN and MCL, and adults with unresectable or metastatic GIST harboring a PDGFRA exon 18 mutation, including PDGFRA D842V mutations.

Product Name : Ayvakit

Product Type : Other Small Molecule

Upfront Cash : Inapplicable

March 02, 2023

blank

Details:

Ayvakit (avapritinib) is a kinase inhibitor approved by the FDA for the treatment of adults with Advanced SM, ASM, SM-AHN and MCL, and adults with unresectable or metastatic GIST harboring a PDGFRA exon 18 mutation, including PDGFRA D842V mutations.


Lead Product(s): Avapritinib

Therapeutic Area: Hematology Brand Name: Ayvakit

Study Phase: Approved FDFProduct Type: Other Small Molecule

Sponsor: Inapplicable

Deal Size: Inapplicable Upfront Cash: Inapplicable

Deal Type: Inapplicable February 14, 2023

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05

India Med Expo
Not Confirmed
India Med Expo
Not Confirmed

Details : Ayvakit (avapritinib) is a kinase inhibitor approved by the FDA for the treatment of adults with Advanced SM, ASM, SM-AHN and MCL, and adults with unresectable or metastatic GIST harboring a PDGFRA exon 18 mutation, including PDGFRA D842V mutations.

Product Name : Ayvakit

Product Type : Other Small Molecule

Upfront Cash : Inapplicable

February 14, 2023

blank

Details:

Ayvakit (avapritinib) is a kinase inhibitor approved by the FDA for the treatment of adults with Advanced SM, ASM, SM-AHN and MCL, and adults with unresectable or metastatic GIST harboring a PDGFRA exon 18 mutation, including PDGFRA D842V mutations.


Lead Product(s): Avapritinib

Therapeutic Area: Hematology Brand Name: Ayvakit

Study Phase: Phase IIIProduct Type: Other Small Molecule

Sponsor: Inapplicable

Deal Size: Inapplicable Upfront Cash: Inapplicable

Deal Type: Inapplicable January 23, 2023

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06

India Med Expo
Not Confirmed
India Med Expo
Not Confirmed

Details : Ayvakit (avapritinib) is a kinase inhibitor approved by the FDA for the treatment of adults with Advanced SM, ASM, SM-AHN and MCL, and adults with unresectable or metastatic GIST harboring a PDGFRA exon 18 mutation, including PDGFRA D842V mutations.

Product Name : Ayvakit

Product Type : Other Small Molecule

Upfront Cash : Inapplicable

January 23, 2023

blank

Details:

AYVAKIT (avapritinib) is a kinase inhibitor approved by the FDA for the treatment of adults with Advanced SM, including aggressive SM (ASM), SM with an associated hematological neoplasm (SM-AHN) and mast cell leukemia (MCL).


Lead Product(s): Avapritinib

Therapeutic Area: Hematology Brand Name: Ayvakit

Study Phase: Phase IIIProduct Type: Other Small Molecule

Sponsor: Inapplicable

Deal Size: Inapplicable Upfront Cash: Inapplicable

Deal Type: Inapplicable November 22, 2022

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07

India Med Expo
Not Confirmed
India Med Expo
Not Confirmed

Details : AYVAKIT (avapritinib) is a kinase inhibitor approved by the FDA for the treatment of adults with Advanced SM, including aggressive SM (ASM), SM with an associated hematological neoplasm (SM-AHN) and mast cell leukemia (MCL).

Product Name : Ayvakit

Product Type : Other Small Molecule

Upfront Cash : Inapplicable

November 22, 2022

blank

Details:

Ayvakit (avapritinib) is a FDA approved kinase inhibitor for treatment adults with Advanced SM, ASM, SM-AHN and MCL, also for adults with GIST harboring a PDGFRA exon 18 mutation, including PDGFRA D842V mutations.


Lead Product(s): Avapritinib

Therapeutic Area: Hematology Brand Name: Ayvakit

Study Phase: Phase IIProduct Type: Other Small Molecule

Sponsor: Inapplicable

Deal Size: Inapplicable Upfront Cash: Inapplicable

Deal Type: Inapplicable November 12, 2022

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08

India Med Expo
Not Confirmed
India Med Expo
Not Confirmed

Details : Ayvakit (avapritinib) is a FDA approved kinase inhibitor for treatment adults with Advanced SM, ASM, SM-AHN and MCL, also for adults with GIST harboring a PDGFRA exon 18 mutation, including PDGFRA D842V mutations.

Product Name : Ayvakit

Product Type : Other Small Molecule

Upfront Cash : Inapplicable

November 12, 2022

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Details:

Primary endpoint of PIONEER trial of AYVAKIT (Avapritinib) in non-advanced SM to be updated to mean change in total symptom score, previously a key secondary endpoint, based on U.S. FDA recommendation.


Lead Product(s): Avapritinib

Therapeutic Area: Hematology Brand Name: Ayvakit

Study Phase: Approved FDFProduct Type: Other Small Molecule

Sponsor: Inapplicable

Deal Size: Inapplicable Upfront Cash: Inapplicable

Deal Type: Inapplicable September 06, 2022

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09

India Med Expo
Not Confirmed
India Med Expo
Not Confirmed

Details : Primary endpoint of PIONEER trial of AYVAKIT (Avapritinib) in non-advanced SM to be updated to mean change in total symptom score, previously a key secondary endpoint, based on U.S. FDA recommendation.

Product Name : Ayvakit

Product Type : Other Small Molecule

Upfront Cash : Inapplicable

September 06, 2022

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Details:

The trial, which was designed to assess AYVAKIT (avapritinib) plus best available care versus placebo plus best available care (control arm), achieved its primary endpoint with a highly significant difference in the mean change in total symptom score at 24 weeks (p=0.003).


Lead Product(s): Avapritinib

Therapeutic Area: Hematology Brand Name: Ayvakit

Study Phase: Approved FDFProduct Type: Other Small Molecule

Sponsor: Inapplicable

Deal Size: Inapplicable Upfront Cash: Inapplicable

Deal Type: Inapplicable August 17, 2022

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10

India Med Expo
Not Confirmed
India Med Expo
Not Confirmed

Details : The trial, which was designed to assess AYVAKIT (avapritinib) plus best available care versus placebo plus best available care (control arm), achieved its primary endpoint with a highly significant difference in the mean change in total symptom score at ...

Product Name : Ayvakit

Product Type : Other Small Molecule

Upfront Cash : Inapplicable

August 17, 2022

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INTERMEDIATE SUPPLIERS

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LinkChem

China
India Med Expo
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LinkChem

China
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CAS Number : 89793-12-4

End Use API : Avapritinib

About The Company : LinkChem is a leading China headquartered CMO | CRO provider within the pharmaceutical industry. Our core focus includes: custom synthesis, process development,...

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FDF Dossiers

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India Med Expo
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AVAPRITINIB

Brand Name : AYVAKIT

Dosage Form : TABLET;ORAL

Dosage Strength : 100MG

Packaging :

Approval Date : 2020-01-09

Application Number : 212608

Regulatory Info : RX

Registration Country : USA

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India Med Expo
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India Med Expo
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AVAPRITINIB

Brand Name : AYVAKIT

Dosage Form : TABLET;ORAL

Dosage Strength : 200MG

Packaging :

Approval Date : 2020-01-09

Application Number : 212608

Regulatory Info : RX

Registration Country : USA

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India Med Expo
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India Med Expo
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AVAPRITINIB

Brand Name : AYVAKIT

Dosage Form : TABLET;ORAL

Dosage Strength : 300MG

Packaging :

Approval Date : 2020-01-09

Application Number : 212608

Regulatory Info : RX

Registration Country : USA

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India Med Expo
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AVAPRITINIB

Brand Name : AYVAKIT

Dosage Form : TABLET;ORAL

Dosage Strength : 25MG

Packaging :

Approval Date : 2021-06-16

Application Number : 212608

Regulatory Info : RX

Registration Country : USA

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05

India Med Expo
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India Med Expo
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AVAPRITINIB

Brand Name : AYVAKIT

Dosage Form : TABLET;ORAL

Dosage Strength : 50MG

Packaging :

Approval Date : 2021-06-16

Application Number : 212608

Regulatory Info : RX

Registration Country : USA

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06

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India Med Expo
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AVAPRITINIB

Brand Name : AYVAKYT

Dosage Form : TABLET

Dosage Strength : 25MG

Packaging :

Approval Date :

Application Number : 2551764

Regulatory Info : Prescription

Registration Country : Canada

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India Med Expo
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AVAPRITINIB

Brand Name : AYVAKYT

Dosage Form : TABLET

Dosage Strength : 50MG

Packaging :

Approval Date :

Application Number : 2551772

Regulatory Info : Prescription

Registration Country : Canada

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India Med Expo
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AVAPRITINIB

Brand Name : AYVAKYT

Dosage Form : TABLET

Dosage Strength : 100MG

Packaging :

Approval Date :

Application Number : 2551780

Regulatory Info : Prescription

Registration Country : Canada

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India Med Expo
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AVAPRITINIB

Brand Name : AYVAKYT

Dosage Form : TABLET

Dosage Strength : 200MG

Packaging :

Approval Date :

Application Number : 2551799

Regulatory Info : Prescription

Registration Country : Canada

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FDA okays 50 new drugs in 2024; BMS’ Cobenfy, Lilly’s Kisunla lead pack of breakthrough therapies
In 2024, the biopharma industry continued to advance on its robust trajectory of innovation. Though the US Food and Drug Administration’s Center for Drug Evaluation and Research (CDER) and the Center for Biologics Evaluation and Research (CBER) approved fewer drugs, there was a significant increase in medical breakthroughs.While the CDER approved 50 new drugs in 2024, as compared to 55 in 2023, the CBER granted 14 biologics approvals in 2024, down from 20 in 2023.The European Medicines Agency (EMA) approved 34 new therapies, up from 32 in 2023, while Health Canada granted 28 approvals, down from 38 in 2023.The year saw long-awaited treatments being approved in areas such as schizophrenia and Alzheimer’s disease in the second half (H2) of 2024. In H1 2024, drugs to treat metabolic dysfunction-associated steatohepatitis (MASH) and chronic obstructive pulmonary disease (COPD) had been granted FDA approvals.As the year drew to a close, FDA began approving drugs at a feverish pace, with 29 of the CDER’s 50 approvals coming in H2.Like most years, the landscape of drug approvals was dominated by oncology, with 15 of the 50 drugs (30 percent) approved targeting various forms of cancer. This was followed by dermatology and non-malignant hematology, each accounting for 12 percent of approvals. Notably, small molecules continued to dominate the market, making up for 64 percent of the new drug approvals, while 32 percent were proteins, including monoclonal and bi-specific antibodies. View New Drug Approvals in 2024 with Estimated Sales (Free Excel Available)Karuna-BMS’ schizophrenia drug, Lilly’s Alzheimer’s med, Neurocrine’s Crenessity dominate list of pathbreaking approvals in H2Out of the 50 new drugs approved in 2024, CDER identified 24 (48 percent) as first-in-class, showcasing novel mechanisms of action. The most anticipated approval of 2024 was Karuna and Bristol Myers Squibb’s Cobenfy, a groundbreaking treatment for schizophrenia. This fixed-dose combination of xanomeline and trospium chloride represents the first novel mechanism of action in decades for this debilitating psychiatric condition. Analysts forecast peak annual sales of over US$ 3.3 billion for Cobenfy. Eli Lilly’s Alzheimer’s drug Kisunla (donanemab) became the third amyloid-targeting antibody to gain FDA approval. Unlike its predecessors, Kisunla offers a unique limited-duration treatment regimen, allowing patients to discontinue therapy once amyloid levels in the brain drop below a certain threshold. Priced at approximately US$ 32,000 per year, it is positioned as a cost-effective alternative to existing treatments. Analysts estimate peak sales of US$ 2.4 billion for Kisunla.Crenessity (crinecerfont), developed by Neurocrine Biosciences, became the first FDA-approved treatment in decades for classic congenital adrenal hyperplasia (genetic conditions that affect the adrenal glands). Similarly, Vertex’s triple combination therapy of deutivacaftor, tezacaftor & vanzacaftor (Alyftrek) for cystic fibrosis represents a significant advancement in genetic disease treatment. Analysts forecast peak sales exceeding US$ 8.3 billion, underscoring the therapy’s potential to transform patient care.Meanwhile, Bridgebio’s Attruby (acoramidis hydrochloride) emerged as a promising treatment for cardiac amyloidosis, a life-threatening condition. View New Drug Approvals in 2024 with Estimated Sales (Free Excel Available) Roche’s Itovebi, Checkpoint’s Unloxcyt clinch FDA approvals in H2 2024; forecast to achieve blockbuster statusThe dominance of cancer drug approvals reflects the ongoing focus on targeted therapies, immuno-oncology, and precision medicine to improve outcomes for patients with hard-to-treat cancers.Among the year’s notable FDA approvals was Genentech’s Itovebi (inavolisib), another targeted therapy that treats hormone receptor-positive (HR+), HER2-negative breast cancer. Itovebi is a PI3Kα inhibitor designed specifically for patients with PIK3CA mutations, a common driver of resistance to endocrine therapy in breast cancer. It demonstrated a more tolerable safety profile. Roche projects Itovebi’s peak (annual) sales to reach CHF 2 billion (US$ 2.3 billion).Checkpoint Therapeutics’ Unloxcyt (cosibelimab) joined the crowded checkpoint inhibitor market as the eleventh PD-1/PD-L1-targeting monoclonal antibody approved by the FDA. It was granted approval for cutaneous squamous cell carcinoma (cSCC), an aggressive form of skin cancer with high recurrence rates. As compared to other checkpoint inhibitors, like Keytruda (pembrolizumab) and Opdivo (nivolumab), Unloxcyt is likely to offer an advantage in immune activation.FDA also approved Astellas’ Vyloy (zolbetuximab), a first-in-class monoclonal antibody for metastatic gastric and gastroesophageal junction (GEJ) adenocarcinoma. Analysts forecast peak sales of approximately US$ 850 million for Vyloy.Syndax Pharmaceuticals’ Revuforj (revumenib) was approved by FDA to treat a type of acute leukemia in both adults and children. This approval introduces a novel class of medications known as menin inhibitors. These agents are currently in clinical development for the treatment of genetically defined subsets of acute leukemia. These inhibitors function by preventing the activation of cancer growth-related proteins. View New Drug Approvals in 2024 with Estimated Sales (Free Excel Available) Potential blockbusters Lilly’s Ebglyss, Galderma’s Nemluvio lead advances in dermatologyEli Lilly’s Ebglyss (lebrikizumab) garnered significant attention. Approved by FDA for moderate-to-severe atopic dermatitis, this monoclonal antibody introduces a less burdensome dosing regimen compared to its competitors, with maintenance therapy required only once a month. This feature positions it as a potential contender to Dupixent (dupilumab), a market leader in atopic dermatitis. Ebglyss sales are forecast to reach US$ 1.9 billion by 2030.Galderma’s Nemluvio (nemolizumab) secured FDA approval for two indications in 2024 — prurigo nodularis (a chronic disorder of the skin) and moderate-to-severe atopic dermatitis in patients aged 12 years and older. As the first humanized IgG2 monoclonal antibody targeting the IL-31 receptor, Nemluvio directly inhibits the key driver of itch and inflammation in both these conditions. With its unique mechanism and broad dermatology potential, analysts forecast peak sales of approximately US$ 1.66 billion. Ebglyss and Nemluvio underscore the growing importance of biologics in dermatological care.Botanix Pharmaceuticals also made strides in dermatology by clinching an FDA approval for Sofdra (sofpironium) in June. The drug has been okayed for the treatment of primary axillary hyperhidrosis, a condition characterized by excessive sweating.Ascendis Pharma’s Yorvipath (palopegteriparatide), a therapy approved by FDA to treat hypoparathyroidism, is forecast to achieve blockbuster sales of US$ 1.8 billion by 2030, highlighting its potential to transform endocrine care. View New Drug Approvals in 2024 with Estimated Sales (Free Excel Available) Our viewOverall, 2024 was defined by its breakthrough drug approvals. The year also saw significant reduction in complete response letters (CRLs) — they dropped from 43 in 2023 to just 29 in 2024. This suggests improved industry preparedness and alignment with regulatory expectations.The new year began with the approval of Datroway (datopotamab deruxtecan) from AstraZeneca and Daiichi Sankyo, marking a significant advancement in oncology. Several other promising new drugs are coming up for FDA approval this year, such as J&J’s nipocalimab, Vertex Pharmaceuticals’ suzetrigine, Elevar Therapeutics’ rivoceranib/camrelizumab, Sanofi’s fitusiran and GSK’s gepotidacin. Hopefully, the momentum of breakthrough approvals will continue through 2025, political headwinds in the US notwithstanding. 

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https://www.pharmacompass.com/radio-compass-blog/fda-okays-50-new-drugs-in-2024-bms-cobenfy-lilly-s-kisunla-lead-pack-of-breakthrough-therapies

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30 Jan 2025

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Looking for 1703793-34-3 / Avapritinib API manufacturers, exporters & distributors?

Avapritinib manufacturers, exporters & distributors 1

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PharmaCompass offers a list of Avapritinib API manufacturers, exporters & distributors, which can be sorted by GMP, USDMF, JDMF, KDMF, CEP (COS), WC, Price,and more, enabling you to easily find the right Avapritinib manufacturer or Avapritinib supplier for your needs.

Send us enquiries for free, and we will assist you in establishing a direct connection with your preferred Avapritinib manufacturer or Avapritinib supplier.

PharmaCompass also assists you with knowing the Avapritinib API Price utilized in the formulation of products. Avapritinib API Price is not always fixed or binding as the Avapritinib Price is obtained through a variety of data sources. The Avapritinib Price can also vary due to multiple factors, including market conditions, regulatory modifications, or negotiated pricing deals.

API | Excipient name

Avapritinib

Synonyms

Blu-285, 1703793-34-3, Ayvakit, Avapritinib [inn], Blu285, (1s)-1-(4-fluorophenyl)-1-[2-[4-[6-(1-methylpyrazol-4-yl)pyrrolo[2,1-f][1,2,4]triazin-4-yl]piperazin-1-yl]pyrimidin-5-yl]ethanamine

Cas Number

1703793-34-3

Unique Ingredient Identifier (UNII)

513P80B4YJ

About Avapritinib

Avapritinib is an orally bioavailable inhibitor of specific mutated forms of platelet-derived growth factor receptor alpha (PDGFR alpha; PDGFRa) and mast/stem cell factor receptor c-Kit (SCFR), with potential antineoplastic activity. Upon oral administration, avapritinib specifically binds to and inhibits specific mutant forms of PDGFRa and c-Kit, including the PDGFRa D842V mutant and various KIT exon 17 mutants. This results in the inhibition of PDGFRa- and c-Kit-mediated signal transduction pathways and the inhibition of proliferation in tumor cells that express these PDGFRa and c-Kit mutants. PDGFRa and c-Kit, protein tyrosine kinases and tumor-associated antigens (TAAs), are mutated in various tumor cell types; they play key roles in the regulation of cellular proliferation.

BLU-285 (Avapritinib) Manufacturers

A BLU-285 (Avapritinib) manufacturer is defined as any person or entity involved in the manufacture, preparation, processing, compounding or propagation of BLU-285 (Avapritinib), including repackagers and relabelers. The FDA regulates BLU-285 (Avapritinib) manufacturers to ensure that their products comply with relevant laws and regulations and are safe and effective to use. BLU-285 (Avapritinib) API Manufacturers are required to adhere to Good Manufacturing Practices (GMP) to ensure that their products are consistently manufactured to meet established quality criteria.

click here to find a list of BLU-285 (Avapritinib) manufacturers with USDMF, JDMF, KDMF, CEP, GMP, COA and API Price related information on PhamaCompass.

BLU-285 (Avapritinib) Suppliers

A BLU-285 (Avapritinib) supplier is an individual or a company that provides BLU-285 (Avapritinib) active pharmaceutical ingredient (API) or BLU-285 (Avapritinib) finished formulations upon request. The BLU-285 (Avapritinib) suppliers may include BLU-285 (Avapritinib) API manufacturers, exporters, distributors and traders.

click here to find a list of BLU-285 (Avapritinib) suppliers with USDMF, JDMF, KDMF, CEP, GMP, COA and API Price related information on PharmaCompass.

BLU-285 (Avapritinib) GMP

BLU-285 (Avapritinib) Active pharmaceutical ingredient (API) is produced in GMP-certified manufacturing facility.

GMP stands for Good Manufacturing Practices, which is a system used in the pharmaceutical industry to make sure that goods are regularly produced and monitored in accordance with quality standards. The FDA’s current Good Manufacturing Practices requirements are referred to as cGMP or current GMP which indicates that the company follows the most recent GMP specifications. The World Health Organization (WHO) has its own set of GMP guidelines, called the WHO GMP. Different countries can also set their own guidelines for GMP like China (Chinese GMP) or the EU (EU GMP).

PharmaCompass offers a list of BLU-285 (Avapritinib) GMP manufacturers, exporters & distributors, which can be sorted by USDMF, JDMF, KDMF, CEP (COS), WC, API price, and more, enabling you to easily find the right BLU-285 (Avapritinib) GMP manufacturer or BLU-285 (Avapritinib) GMP API supplier for your needs.

BLU-285 (Avapritinib) CoA

A BLU-285 (Avapritinib) CoA (Certificate of Analysis) is a formal document that attests to BLU-285 (Avapritinib)'s compliance with BLU-285 (Avapritinib) specifications and serves as a tool for batch-level quality control.

BLU-285 (Avapritinib) CoA mostly includes findings from lab analyses of a specific batch. For each BLU-285 (Avapritinib) CoA document that a company creates, the USFDA specifies specific requirements, such as supplier information, material identification, transportation data, evidence of conformity and signature data.

BLU-285 (Avapritinib) may be tested according to a variety of international standards, such as European Pharmacopoeia (BLU-285 (Avapritinib) EP), BLU-285 (Avapritinib) JP (Japanese Pharmacopeia) and the US Pharmacopoeia (BLU-285 (Avapritinib) USP).

Inform the supplier about your product requirements, specifying if you need a product with particular monograph like EP (Ph. Eur.), USP, JP, BP, or any other quality. In addition, clarify whether you need hydrochloride (HCl), anhydricum, base, micronisatum or a specific level of purity. To find reputable suppliers, utilize the filters and select those certified by GMP, FDA, or any other certification as per your requirement.
For your convenience, we have listed synonyms and CAS numbers to help you find the best supplier. The use of synonyms and CAS numbers can be helpful in identifying potential suppliers, but it is crucial to note that they might not always indicate the exact same product. It is important to confirm the product details with the supplier before making a purchase to ensure that it meets your requirements.
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