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Blueprint Medicines Corporation — Rare Disease Pipeline | Kisho | Kisho

Blueprint Medicines Corporation

Other

4

Orphan Designations

1

FDA Approvals

17

Rare Diseases

0

News Articles

Disease	Drug(s)	Designation	Approved
acute mast cell leukemia	Ayvakit	Orphan Designation	-
adult embryonal tumor with multilayered rosettes, c19mc-altered	Ayvakit	Orphan Designation	-
adult hepatocellular carcinoma	fisogatinib	Orphan Designation	-
adult neuronal ceroid lipofuscinosis	Ayvakit	Orphan Designation	-
adult pleomorphic rhabdomyosarcoma	Ayvakit	Orphan Designation	-
adult-onset chronic progressive external ophthalmoplegia with mitochondrial myopathy	Ayvakit	Orphan Designation	-
adult-onset non-insulinoma persistent hyperinsulinemic hypoglycemia	Ayvakit	Orphan Designation	-
adult-onset proximal spinal muscular atrophy, autosomal dominant	Ayvakit	Orphan Designation	-
cataract 13 with adult I phenotype	Ayvakit	Orphan Designation	-
chronic mast cell leukemia	Ayvakit	Orphan Designation	-
combined hepatocellular carcinoma and cholangiocarcinoma	fisogatinib	Orphan Designation	-
gastrointestinal stromal tumor	Ayvakit	Orphan Designation	-
hepatocellular carcinoma	fisogatinib	Orphan Designation	-
mast cell leukemia	Ayvakit	Orphan Designation	-
mastocytosis	Selective KIT InhibitorAyvakit	Orphan Designation	-
nut midline carcinoma	Ayvakit	Orphan Designation	-
primary adult heart tumor	Ayvakit	Orphan Designation	-

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