An expansive pipeline
We are focused on the rapid design and development of precision therapies for people with cancer and rare diseases.
Our 2020 Blueprint
2
marketed products
4
pending
marketing
applications
marketing
applications
6
clinical-stage
therapeutic
candidates
therapeutic
candidates
8
research
programs
programs
Our “2020 Blueprint” strategy establishes a planned path to transform Blueprint Medicines, by the end of 2020, into a global commercial enterprise focused on delivering a portfolio of precision therapies to patients with cancer and rare diseases. See our press release.
Rapidly advancing pipeline of investigational precision therapies
| 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
|
3Late-stage development | |
|
»PDGFRA GIST
1,2
4 - Regulatory submission
|
NDA/MAA
|
4Regulatory submission |
|
»4L GIST
1,2
3 - Late-stage development
|
NDA/MAA
|
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
3 - Late-stage development
|
NDA
|
3Late-stage development |
|
»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 |
|
»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 (+CS-1001)
2
0 - None
|
|
0None |
|
Research program: 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 3
1 - Discovery
|
Research program: MAP4K1
|
1Discovery |
|
Research programs: 3 undisclosed targets 3
1 - Discovery
|
Research programs: 3 undisclosed targets
|
1Discovery |
ongoing or completed
planned
Updated as of November 5, 2019.
- 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.
- 3In 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. MTC = medullary thyroid cancer. NSCLC = non-small cell lung cancer. SM = systemic mastocytosis.