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Alnylam Pharmaceuticals, Inc. — Rare Disease Pipeline | Kisho | Kisho

Alnylam Pharmaceuticals, Inc.

Other

6

Orphan Designations

4

FDA Approvals

18

Rare Diseases

1

News Articles

Disease	Drug(s)	Designation	Approved
ATTRV30M amyloidosis	RNA interference (RNAi) therapeutic, that targets the expression of TTRAmvuttrarevusiranONPATTRO	Orphan Designation	-
adult embryonal tumor with multilayered rosettes, c19mc-altered	GIVLAARI	Orphan Designation	-
adult neuronal ceroid lipofuscinosis	Oxlumo	Orphan Designation	-
adult pleomorphic rhabdomyosarcoma	Oxlumo	Orphan Designation	-
adult-onset chronic progressive external ophthalmoplegia with mitochondrial myopathy	GIVLAARI	Orphan Designation	-
agenesis of the corpus callosum with peripheral neuropathy	AmvuttraONPATTRO	Orphan Designation	-
cataract 13 with adult I phenotype	GIVLAARI	Orphan Designation	-
familial amyloid neuropathy	RNA interference (RNAi) therapeutic, that targets the expression of TTRAmvuttrarevusiranONPATTRO	Orphan Designation	-
hepatic porphyria	GIVLAARI	Orphan Designation	-
hereditary spastic paraplegia 11	AmvuttraONPATTRO	Orphan Designation	-
hereditary spastic paraplegia 61	AmvuttraONPATTRO	Orphan Designation	-
nut midline carcinoma	GIVLAARI	Orphan Designation	-
polyneuropathy	ONPATTRO	-	FDA Approved
polyneuropathy-hand defect syndrome	AmvuttraONPATTRO	Orphan Designation	-
primary adult heart tumor	Oxlumo	Orphan Designation	-
primary biliary cholangitis	Oxlumo	Orphan Designation	-
primary hyperoxaluria	Oxlumo	Orphan Designation	-
primary hyperoxaluria type 1	Oxlumo	Orphan Designation	-

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