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
AYVAKIT™ (avapritinib): PDGFRA
5Approved
»PDGFRA GIST 1,2,3
5 - Approved
5Approved
GAVRETO® (pralsetinib): RET
3Late-stage development
»RET+ NSCLC 1,2,4,5
5 - Approved
5Approved
»RET+ thyroid cancer 1,2,4,6
5 - Approved
MAA
5Approved
»Other RET-altered solid tumors 1,2,4
2 - Early-stage development
2Early-stage development
Fisogatinib: FGFR4
2Early-stage development
»Advanced HCC (+/- sugemalimab) 2
2 - Early-stage development
2Early-stage development
BLU-701: EGFR double mutant
0None
»EGFR+ NSCLC 1,7
1 - Discovery
1Discovery
BLU-945: EGFR triple mutant
0None
»EGFR+ NSCLC 1,7
2 - Early-stage development
2Early-stage development
BLU-222: CDK2
2Early-stage development
»Cyclin E-aberrant cancers
1 - Discovery
1Discovery
Multiple research programs
1 - Discovery
Multiple research programs
1Discovery
Hematologic disorders
Hematologic disorders Molecular target Clinical phase
Hematologic disorders
Hematologic disorders Multiple research programs
Discovery
Early stage development
Late stage development
Regulatory submission
Approved
test
AYVAKIT: KIT
3Late-stage development
»Advanced SM 2,8
5 - Approved
MAA
5Approved
»Non-advanced SM 2
3 - Late-stage development
3Late-stage development
BLU-263: KIT
0None
»Non-advanced SM
3 - Late-stage development
3Late-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
BLU-852: MAP4K1 9
0None
»Advanced cancers
1 - Discovery
1Discovery
Research program 9
1 - Discovery
Research program
1Discovery

ongoing or completed

planned

Updated as of November 9, 2021.

  • 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 under the brand name AYVAKYT® 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.
  • 6Received accelerated approval in the U.S. for the treatment of patients with advanced or metastatic RET-mutant medullary thyroid cancer and RET fusion-positive thyroid cancer. Continued approval may be contingent on confirmatory trials.
  • 7Zai Lab has exclusive rights to develop and commercialize BLU-701 and BLU-945 in Mainland China, Hong Kong, Macau and Taiwan.
  • 8Approved in the U.S. for the treatment of adults with advanced SM, including aggressive SM, SM with an associated hematologic neoplasm and mast cell leukemia.
  • 9In collaboration with Roche. For one of the programs, Blueprint Medicines has U.S. commercial rights and Roche has ex-U.S. commercialization rights. For one of the programs, Roche has worldwide commercialization rights.

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

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