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
6 clinical-stage
therapeutic
candidates
8 research
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.

Our portfolio

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: PDGFRA 1 »Advanced PDGFRA mutant GIST
3 - Late-stage development
Avapritinib: PDGFRA
NDA/MAA submitted
3Late-stage development
Avapritinib: KIT and PDGFRA 1 »Advanced 4L GIST
3 - Late-stage development
Avapritinib: KIT and PDGFRA
NDA/MAA submitted
3Late-stage development
Avapritinib: KIT and PDGFRA 1 »Advanced 3L GIST
3 - Late-stage development
Avapritinib: KIT and PDGFRA
NDA planned 2020
3Late-stage development
Avapritinib: KIT and PDGFRA 1 »Advanced 2L GIST
2 - Early-stage development
Avapritinib: KIT and PDGFRA
Trial planned 2H 2019
2Early-stage development
Pralsetinib (BLU-667): RET 1 »Advanced 2L RET-fusion NSCLC
3 - Late-stage development
Pralsetinib (BLU-667): RET
NDA planned Q1 2020
3Late-stage development
Pralsetinib: RET 1 »Advanced 1L RET-fusion NSCLC
0 - None
Pralsetinib: RET
Trial planned 2H 2019
0None
Pralsetinib: RET 1 »Advanced EGFR-mutant NSCLC (+osimertinib)
0 - None
Pralsetinib: RET
Trial planned 2H 2019
0None
Pralsetinib: RET 1 »Advanced 2L RET-mutant MTC
3 - Late-stage development
Pralsetinib: RET
NDA planned 1H 2020
3Late-stage development
Pralsetinib: RET 1 »Other RET-altered solid tumors
2 - Early-stage development
Pralsetinib: RET
2Early-stage development
Fisogatinib (BLU-554): FGFR4 1 »Advanced HCC
2 - Early-stage development
Fisogatinib (BLU-554): FGFR4
2Early-stage development
Fisogatinib 1 »Advanced HCC (+CS-1001)
0 - None
Fisogatinib
Trial planned 2H 2019
0None
4 WHOLLY OWNED PROGRAMS »Undisclosed Targets
1 - Discovery
4 WHOLLY OWNED PROGRAMS
1Discovery
Rare diseases
Rare diseases Molecular target Clinical phase
Rare diseases
Rare diseases 4 WHOLLY OWNED PROGRAMS
Discovery
Early stage development
Late stage development
Regulatory submission
Approved
test
Avapritinib: KIT D816V 1 »Advanced systemic mastocytosis
3 - Late-stage development
Avapritinib: KIT D816V
NDA planned Q1 2020
3Late-stage development
Avapritinib: KIT D816V 1 »Indolent and smoldering systemic mastocytosis
3 - Late-stage development
Avapritinib: KIT D816V
3Late-stage development
BLU-782: ALK2 2 »Fibrodysplasia ossificans progressiva
2 - Early-stage development
BLU-782: ALK2
2Early-stage development
Cancer immunotherapy
Cancer immunotherapy Molecular target Clinical phase
Cancer immunotherapy
Cancer immunotherapy BLU-782: ALK2
Discovery
Early stage development
Late stage development
Regulatory submission
Approved
test
UP TO 5 PROGRAMS 3 »Undisclosed targets
1 - Discovery
UP TO 5 PROGRAMS
1Discovery

Updated as of August 1, 2019

  • 1CStone 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.
  • 2Phase 1 trial in healthy volunteers ongoing; phase 2 trial in patients with FOP planned Q4 2019.
  • 3In collaboration with Roche. Blueprint Medicines has U.S. commercial rights for up to two programs. Roche has worldwide commercialization rights for up to three programs and ex-U.S. commercialization rights for up to two programs.

Definitions: 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.

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