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
| 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
|
5Approved | |
|
»PDGFRA Exon 18 GIST
1,2,3
5 - Approved
|
|
5Approved |
|
»4L GIST
1,2
3 - Late-stage development
|
NDA
|
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
1,2,4
4 - Regulatory submission
|
NDA / MAA
|
4Regulatory submission |
|
»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 |
|
»1L MTC
1,2
2 - Early-stage development
|
|
2Early-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 (+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
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 5
1 - Discovery
|
Research program: MAP4K1
|
1Discovery |
|
Research programs: 3 undisclosed targets 5
1 - Discovery
|
Research programs: 3 undisclosed targets
|
1Discovery |
ongoing or completed
planned
Updated as of April 1, 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. Blueprint Medicines retains all rights in the rest of the world.
- 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. The proposed MAA indication is unresectable or metastatic GIST harboring a PDGFRA D842V mutation.
- 4NDA submitted to FDA in March 2020; plan to submit MAA to EMA in Q2 2020.
- 5In 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. MAA = Marketing Authorization Application. MTC = medullary thyroid cancer. NDA = New Drug Application. NSCLC = non-small cell lung cancer. SM = systemic mastocytosis.
Not for promotional use.