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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