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

Apellis Pharmaceuticals, Inc.

Other

5

Orphan Designations

2

FDA Approvals

18

Rare Diseases

0

News Articles

Disease	Drug(s)	Designation	Approved
Gaucher disease type III	Empaveli	Orphan Designation	-
Sandhoff disease, adult form	Empaveli	Orphan Designation	-
adult neuronal ceroid lipofuscinosis	Empaveli	Orphan Designation	-
adult pleomorphic rhabdomyosarcoma	Empaveli	Orphan Designation	-
adult-onset non-insulinoma persistent hyperinsulinemic hypoglycemia	Empaveli	Orphan Designation	-
adult-onset proximal spinal muscular atrophy, autosomal dominant	Empaveli	Orphan Designation	-
amyotrophic lateral sclerosis	Pegcetacoplan	Orphan Designation	-
ataxia and polyneuropathy, adult-onset	Empaveli	Orphan Designation	-
autoimmune hemolytic anemia	pegcetacoplan	Orphan Designation	-
deafness, congenital, and adult-onset progressive leukoencephalopathy	Empaveli	Orphan Designation	-
immune complex mediated vasculitis	Empaveli	Orphan Designation	-
immune-complex glomerulonephritis	Empaveli	Orphan Designation	-
immunoglobulin-mediated membranoproliferative glomerulonephritis	Empaveli	Orphan Designation	-
neuropathy, hereditary sensory and autonomic, adult-onset, with anosmia	Empaveli	Orphan Designation	-
non-immunoglobulin-mediated membranoproliferative glomerulonephritis	Empaveli	Orphan Designation	-
paroxysmal nocturnal hemoglobinuria	Empaveli	Orphan Designation	-
primary adult heart tumor	Empaveli	Orphan Designation	-
type III hypersensitivity disease	Empaveli	Orphan Designation	-

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