An expansive pipeline

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

Rapidly advancing pipeline

Genomically defined cancers
Genomically defined cancers Molecular target Clinical phase
Genomically defined cancers
Genomically defined cancers
Discovery
Early stage development
Late stage development
Regulatory submission
Approved
test
Avapritinib: PDGFRA
5Approved
»PDGFRA GIST 1,2,3
5 - Approved
5Approved
Pralsetinib: RET
3Late-stage development
»RET+ NSCLC 1,2,4,5
5 - Approved
5Approved
»EGFR+ NSCLC (+osimertinib) 1,2,4
0 - None
0None
»RET+ MTC 1,2,4
3 - Late-stage development
NDA
3Late-stage development
»RET+ thyroid cancer 1,2,4
4 - Regulatory submission
NDA
4Regulatory submission
»Other RET-altered solid tumors 1,2,4
2 - Early-stage development
2Early-stage development
Fisogatinib: FGFR4
2Early-stage development
»Advanced HCC 2
2 - Early-stage development
2Early-stage development
»Advanced HCC (+CS1001) 2
2 - Early-stage development
2Early-stage development
BLU-945: 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
2 - Early-stage development
2Early-stage development
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 6
1 - Discovery
Research program: MAP4K1
1Discovery
Research programs: 3 undisclosed targets 6
1 - Discovery
Research programs: 3 undisclosed targets
1Discovery

ongoing or completed

planned

Updated as of September 25, 2020.

  • 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.
  • 3Approved in the U.S. for the treatment of adults with unresectable or metastatic GIST harboring a PDGFRA exon 18 mutation, including PDGFRA D842V mutations. Received conditional marketing authorization in Europe for the treatment of adults with unresectable or metastatic GIST harboring the PDGFRA D842V mutation.
  • 4In collaboration with Roche. Blueprint Medicines and Roche have co-exclusive rights to develop and commercialize pralsetinib in the U.S., and Roche has exclusive rights to develop and commercialize pralsetinib outside the U.S., excluding the CStone territory.
  • 5Received accelerated approval in the U.S. for the treatment of adults with metastatic RET fusion-positive NSCLC. Continued approval may be contingent on a confirmatory trial. The proposed indication for the MAA is locally advanced or metastatic RET fusion-positive NSCLC previously treated with platinum-based chemotherapy.
  • 6In 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.

GIST = gastrointestinal stromal tumors. HCC = hepatocellular carcinoma. MAA = marketing authorization application. MTC = medullary thyroid cancer. NDA = new drug application. NSCLC = non-small cell lung cancer. SM = systemic mastocytosis.

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