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Alexion Pharmaceuticals Inc. — Rare Disease Pipeline | Kisho | Kisho

Alexion Pharmaceuticals Inc.

Other

Orphan Designations

FDA Approvals

Rare Diseases

News Articles

Disease	Drug(s)	Designation	Approved
ATTRV30M amyloidosis	recombinant human anti-ATTR-immunoglobulin G1 monoclonal antibody	Orphan Designation	-
Acrodysostosis 1 with or without hormone resistance	ANDEXXA	Orphan Designation	-
Bicervical bicornuate uterus with patent cervix and vagina	KanumaStrensiq	Orphan Designation	-
C3 glomerulonephritis	(2S,4R)-1-(2-(3-acetyl-5-(2-methylpyrimidin-5-yl)-1H-indazol-1-yl)acetyl)-N-(6-bromopyridin-2-yl)-4-fluoropyrrolidine-2-carboxamide	Orphan Designation	-
Char syndrome	Kanuma	Orphan Designation	-
Down syndrome	Strensiq	Orphan Designation	-
Gaucher disease type III	Ultomiris	Orphan Designation	-
Guillain-Barre syndrome	eculizumab	Orphan Designation	-
Miller Fisher syndrome	eculizumab	Orphan Designation	-
Sandhoff disease, adult form	Ultomiris	Orphan Designation	-
Shiga toxin-associated hemolytic uremic syndrome	eculizumab	Orphan Designation	-
acute graft versus host disease	Recombinant human monoclonal antibody against human complement component C5a	Orphan Designation	-
adult neuronal ceroid lipofuscinosis	Ultomiris	Orphan Designation	-
adult pleomorphic rhabdomyosarcoma	Ultomiris	Orphan Designation	-
adult-onset non-insulinoma persistent hyperinsulinemic hypoglycemia	Ultomiris	Orphan Designation	-
adult-onset proximal spinal muscular atrophy, autosomal dominant	Ultomiris	Orphan Designation	-
anosmia for isobutyric acid	ANDEXXA	Orphan Designation	-
ataxia and polyneuropathy, adult-onset	Ultomiris	Orphan Designation	-
atypical hemolytic-uremic syndrome	UltomirisSoliris	Orphan Designation	-
atypical hemolytic-uremic syndrome with B factor anomaly	UltomirisSoliris	Orphan Designation	-
atypical hemolytic-uremic syndrome with MCP/CD46 anomaly	UltomirisSoliris	Orphan Designation	-
atypical hemolytic-uremic syndrome with anti-factor H antibodies	UltomirisSoliris	Orphan Designation	-
autoimmune autonomic ganglionopathy	eculizumab	Orphan Designation	-
bullous pemphigoid	bertilimumab	Orphan Designation	-
carbon monoxide-induced delayed encephalopathy	ravulizumabeculizumabKanumaStrensiq	Orphan Designation	-
coagulation protein disease	ANDEXXA	Orphan Designation	-
deafness, congenital, and adult-onset progressive leukoencephalopathy	Ultomiris	Orphan Designation	-
delayed membranous cranial ossification	ravulizumabeculizumab	Orphan Designation	-
delayed puberty, self-limited	ravulizumabeculizumab	Orphan Designation	-
dermatomyositis	Eculizumab	Orphan Designation	-
disease related to hematopoietic stem cell transplant	Ravulizumab	Orphan Designation	-
double nail for fifth toe	ANDEXXA	Orphan Designation	-
familial amyloid neuropathy	recombinant human anti-ATTR-immunoglobulin G1 monoclonal antibody	Orphan Designation	-
hematopoietic and lymphoid cell neoplasm	Ravulizumab	Orphan Designation	-
hematopoietic and lymphoid system neoplasm	Ravulizumab	Orphan Designation	-
hemoglobin--variants for which the chain carrying the mutation 1S unknown or uncertain	ANDEXXA	Orphan Designation	-
hypophosphatasia	Strensiq	Orphan Designation	-
idiopathic	eculizumab	Orphan Designation	-
idiopathic hypersomnia	eculizumab	Orphan Designation	-
idiopathic membranous glomerulonephritis	eculizumab	Orphan Designation	-
lysosomal acid lipase deficiency	Kanuma	Orphan Designation	-
malignant glioma	selumetinib	Orphan Designation	-
multisystemic smooth muscle dysfunction syndrome	Kanuma	Orphan Designation	-
myasthenia gravis	gefurulimabUltomiris	Orphan Designation	-
natal teeth-intestinal pseudoobstruction-patent ductus syndrome	Kanuma	Orphan Designation	-
neuromyelitis optica	Ultomiris	Orphan Designation	-
neuropathy, hereditary sensory and autonomic, adult-onset, with anosmia	Ultomiris	Orphan Designation	-
nocturnal paroxysmal dystonia	Soliris	Orphan Designation	-
non-immunoglobulin-mediated membranoproliferative glomerulonephritis	(2S,4R)-1-(2-(3-acetyl-5-(2-methylpyrimidin-5-yl)-1H-indazol-1-yl)acetyl)-N-(6-bromopyridin-2-yl)-4-fluoropyrrolidine-2-carboxamide	Orphan Designation	-
paroxysmal nocturnal hemoglobinuria	(1R,3S,5R)-2-(2-(3-acetyl-5-(2-methylpyrimidin-5-yl)-1H-indazol-1-yl)acetyl)-N-(6-bromo-3-methylpyridin-2-yl)-5-methyl-2-azabicyclo[3.1.0]hexane-3-carboxamideVoydeyaUltomirisSoliris	Orphan Designation	-
paroxysmal nocturnal hemoglobinuria 1	Soliris	Orphan Designation	-
paroxysmal nocturnal hemoglobinuria 2	Soliris	Orphan Designation	-
pemphigus	orilanolimab	Orphan Designation	-
primary adult heart tumor	Ultomiris	Orphan Designation	-
primary biliary cholangitis	bis-choline tetrathiomolybdate	Orphan Designation	-
sickle cell disease	a recombinant, humanized single variable domain on a heavy chain (VHH) bispecific antibody that binds with high affinity to human properdin and human serum albumin	Orphan Designation	-
spinal muscular atrophy with congenital bone fractures 1	Strensiq	Orphan Designation	-
spinal muscular atrophy with congenital bone fractures 2	Strensiq	Orphan Designation	-

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