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
Hematologic disorders | ||
---|---|---|
Hematologic disorders | Molecular target | Clinical phase |
Hematologic disorders | ||
Hematologic disorders |
Discovery
Early stage development
Late stage development
Regulatory submission
Approved
|
test |
AYVAKIT® (avapritinib): KIT
|
3Late-stage development | |
»Advanced SM
1,2
5 - Approved
|
|
5Approved |
»Non-advanced SM
1
3 - Late-stage development
|
|
3Late-stage development |
BLU-263: KIT
|
0None | |
»Non-advanced SM
3 - Late-stage development
|
|
3Late-stage development |
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: PDGFRA
|
5Approved | |
»PDGFRA GIST
1,3,4
5 - Approved
|
|
5Approved |
GAVRETO® (pralsetinib): RET
|
3Late-stage development | |
»RET+ NSCLC
1,3,5,6
5 - Approved
|
|
5Approved |
»RET+ thyroid cancer
1,3,5,7
5 - Approved
|
MAA
|
5Approved |
»Other RET+ solid tumors
1,3,5
2 - Early-stage development
|
|
2Early-stage development |
BLU-701: EGFR
|
0None | |
»EGFR+ NSCLC
3,8
2 - Early-stage development
|
|
2Early-stage development |
BLU-945: EGFR
|
0None | |
»EGFR+ NSCLC
3,8
2 - Early-stage development
|
|
2Early-stage development |
BLU-451: EGFR exon 20 insertions
|
0None | |
»EGFR+ NSCLC
3
2 - Early-stage development
|
|
2Early-stage development |
BLU-222: CDK2
|
2Early-stage development | |
»CDK2-vulnerable cancers
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 |
BLU-852: MAP4K1
|
0None | |
»Advanced cancers
9
1 - Discovery
|
|
1Discovery |
Research | ||
---|---|---|
Research | Molecular target | Clinical phase |
Research | ||
Research |
Discovery
Early stage development
Late stage development
Regulatory submission
Approved
|
test |
Multiple undisclosed research programs
1 - Discovery
|
Multiple undisclosed research programs
|
1Discovery |
ongoing or completed
planned
Updated as of May 3, 2022.
- 1CStone Pharmaceuticals has exclusive rights to develop and commercialize avapritinib, pralsetinib and fisogatinib in Mainland China, Hong Kong, Macau and Taiwan.
- 2Approved 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. Received marketing authorization in the European Union under the brand name AYVAKYT® for the treatment of adult patients with aggressive SM, SM with an associated hematological neoplasm or mast cell leukemia, after at least one systemic therapy.
- 3Unresectable or metastatic disease.
- 4Approved 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 the European Union under the brand name AYVAKYT for the treatment of adults with unresectable or metastatic GIST harboring the PDGFRA D842V mutation.
- 5In 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.
- 6Received 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. Received conditional marketing authorization in Europe for the treatment of adults with advanced RET fusion-positive NSCLC not previously treated with a RET inhibitor.
- 7Received 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.
- 8Zai Lab has exclusive rights to develop and commercialize BLU-701 and BLU-945 in Mainland China, Hong Kong, Macau and Taiwan.
- 9In collaboration with Roche. Blueprint Medicines and Roche are conducting activities for up to two programs under the collaboration, including the program targeting MAP4K1. 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. MAA = marketing authorization application. NSCLC = non-small cell lung cancer. SM = systemic mastocytosis.
Blueprint Medicines, AYVAKIT, AYVAKYT, GAVRETO and associated logos are trademarks of Blueprint Medicines Corporation.
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