An expansive pipeline

We are focused on the rapid design and development of precision therapies for people with cancer and rare diseases.

Our 2020 Blueprint

2 marketed products
4 pending
marketing
applications
6 clinical-stage
therapeutic
candidates
8 research
programs

Our “2020 Blueprint” strategy establishes a planned path to transform Blueprint Medicines, by the end of 2020, into a global commercial enterprise focused on delivering a portfolio of precision therapies to patients with cancer and rare diseases. See our press release.

Rapidly advancing pipeline of investigational precision therapies

Genetically defined cancers
Genetically defined cancers Molecular target Clinical phase
Genetically defined cancers
Genetically defined cancers
Discovery
Early stage development
Late stage development
Regulatory submission
Approved
test
Avapritinib: KIT & PDGFRA
3Late-stage development
»PDGFRA GIST 1,2
4 - Regulatory submission
NDA/MAA
4Regulatory submission
»4L GIST 1,2
3 - Late-stage development
NDA/MAA
3Late-stage development
»3L GIST 1,2
3 - Late-stage development
NDA
3Late-stage development
»2L GIST 1,2
2 - Early-stage development
2Early-stage development
Pralsetinib: RET
3Late-stage development
»2L RET+ NSCLC 1,2
3 - Late-stage development
NDA
3Late-stage development
»1L RET+ NSCLC 1,2
3 - Late-stage development
3Late-stage development
»EGFR+ NSCLC (+osimertinib) 1,2
0 - None
0None
»2L MTC 1,2
3 - Late-stage development
NDA
3Late-stage development
»Other RET-altered solid tumors 1,2
2 - Early-stage development
2Early-stage development
Fisogatinib: FGFR4
2Early-stage development
»Advanced HCC 2
2 - Early-stage development
2Early-stage development
»Advanced HCC (+CS-1001) 2
0 - None
0None
Research program: EGFR+ T790M/C797S triple mutant
0None
»EGFR+ NSCLC 1
1 - Discovery
1Discovery
Research program: EGFR+ C797S double mutant
0None
»EGFR+ NSCLC 1
1 - Discovery
1Discovery
Research programs: 2 undisclosed targets
1 - Discovery
Research programs: 2 undisclosed targets
1Discovery
Rare diseases
Rare diseases Molecular target Clinical phase
Rare diseases
Rare diseases Research programs: 2 undisclosed targets
Discovery
Early stage development
Late stage development
Regulatory submission
Approved
test
Avapritinib: KIT
3Late-stage development
»Advanced SM 2
3 - Late-stage development
NDA
3Late-stage development
»Indolent SM 2
3 - Late-stage development
3Late-stage development
BLU-263: KIT
0None
»Indolent SM
1 - Discovery
1Discovery
Cancer immunotherapy
Cancer immunotherapy Molecular target Clinical phase
Cancer immunotherapy
Cancer immunotherapy
Discovery
Early stage development
Late stage development
Regulatory submission
Approved
test
Research program: MAP4K1 3
1 - Discovery
Research program: MAP4K1
1Discovery
Research programs: 3 undisclosed targets 3
1 - Discovery
Research programs: 3 undisclosed targets
1Discovery

ongoing or completed

planned

Updated as of November 5, 2019.

  • 1Unresectable or metastatic disease.
  • 2CStone Pharmaceuticals has exclusive rights to develop and commercialize avapritinib, pralsetinib and fisogatinib in Mainland China, Hong Kong, Macau and Taiwan. Blueprint Medicines retains all rights in the rest of the world.
  • 3In collaboration with Roche. Blueprint Medicines has U.S. commercial rights for up to two programs. Roche has worldwide commercialization rights for up to two programs and ex-U.S. commercialization rights for up to two programs.

1L = first-line. 2L = second-line. 3L = third-line. 4L = fourth-line. GIST = gastrointestinal stromal tumors. HCC = hepatocellular carcinoma. MTC = medullary thyroid cancer. NSCLC = non-small cell lung cancer. SM = systemic mastocytosis.

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