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

Regeneron Pharmaceuticals, Inc.

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A biopharmaceutical company focused on developing treatments for rare diseases, with 8 FDA-approved drugs and 27 orphan drug designations across 64 active rare disease indications.

Orphan Designations

FDA Approvals

Rare Diseases

News Articles

Disease	Drug(s)	Designation	Approved
Alstrom syndrome	vidutolimod	Orphan Designation	-
Angelman syndrome	PraluentEvkeeza	Orphan Designation	-
Cold-induced sweating syndrome 1	Arcalyst	Orphan Designation	-
Ebola hemorrhagic fever	INMAZEB™	Orphan Designation	-
Finnish type amyloidosis	vidutolimod	Orphan Designation	-
GM1 gangliosidosis	Eylea	Orphan Designation	-
Gaucher disease type III	VeopozDupixent	Orphan Designation	-
Huppke-Brendel syndrome	Adeno-Associated Viral Vector Serotype 1 containing the human otoferlin gene	Orphan Designation	-
MALT lymphoma	odronextamab	Orphan Designation	-
Muckle-Wells syndrome	Arcalyst	Orphan Designation	-
Sandhoff disease, adult form	VeopozDupixent	Orphan Designation	-
acute sensory ataxic neuropathy	PraluentEvkeeza	Orphan Designation	-
adult neuronal ceroid lipofuscinosis	LynozyficDupixent	Orphan Designation	-
adult pleomorphic rhabdomyosarcoma	LynozyficDupixent	Orphan Designation	-
adult-onset Still disease	Interleukin-1 Trap	Orphan Designation	-
adult-onset non-insulinoma persistent hyperinsulinemic hypoglycemia	LynozyficDupixent	Orphan Designation	-
adult-onset proximal spinal muscular atrophy, autosomal dominant	LynozyficDupixent	Orphan Designation	-
amyotrophic lateral sclerosis	Ciliary neurotrophic factor	Orphan Designation	-
aortic valve disease 1	PraluentEvkeeza	Orphan Designation	-
aortic valve stenosis	PraluentEvkeeza	Orphan Designation	-
ataxia and polyneuropathy, adult-onset	VeopozDupixent	Orphan Designation	-
autosomal dominant nonsyndromic hearing loss 17	Adeno-Associated Viral Vector Serotype 1 containing the human otoferlin gene	Orphan Designation	-
bullous pemphigoid	Dupixent	Orphan Designation	-
congenital bile acid synthesis defect 1	Arcalyst	Orphan Designation	-
cryopyrin-associated periodic syndrome	Arcalyst	Orphan Designation	-
deafness, congenital, and adult-onset progressive leukoencephalopathy	VeopozDupixent	Orphan Designation	-
diffuse large B-cell lymphoma	human IgG4-based anti-CD20 x anti-CD3 bispecific monoclonal antibody	Orphan Designation	-
dyscalculia	Interleukin-1 Trap	Orphan Designation	-
eosinophilic esophagitis	Dupixent	Orphan Designation	-
extranodal nasal NK/T cell lymphoma	odronextamab	Orphan Designation	-
exudative vitreoretinopathy 1	Eylea	Orphan Designation	-
fatal multiple mitochondrial dysfunctions syndrome	Arcalyst	Orphan Designation	-
fibrodysplasia ossificans progressiva	garetosmab	Orphan Designation	-
follicular lymphoma	humanized IgG4-based anti-CD20 x anti-CD3 bispecific monoclonal antibody	Orphan Designation	-
hemophilia B	A rAAV8 capsid containing a single-stranded DNA template encoding wild-type human coagulation factor IX protein and LNP1265: a lipid nanoparticle encapsulating a Cas9 mRNA and a single guide RNA	Orphan Designation	-
homozygous familial hypercholesterolemia	PraluentEvkeeza	Orphan Designation	-
immunodeficiency 67	rilonacept	Orphan Designation	-
infectious disease	INMAZEB™	Orphan Designation	-
lethal acantholytic epidermolysis bullosa	INMAZEB™	Orphan Designation	-
lipodystrophy	mibavademab	Orphan Designation	-
lymphatic malformation 13	Eylea	Orphan Designation	-
lysinuric protein intolerance	vidutolimod	Orphan Designation	-
mitochondrial non-syndromic sensorineural hearing loss	Adeno-Associated Viral Vector Serotype 1 containing the human otoferlin gene	Orphan Designation	-
multiple mitochondrial dysfunctions syndrome 10	Arcalyst	Orphan Designation	-
myasthenia gravis	pozelimab and cemdisiran combiantion	Orphan Designation	-
mycosis fungoides	odronextamab	Orphan Designation	-
neuropathy, hereditary sensory and autonomic, adult-onset, with anosmia	VeopozDupixent	Orphan Designation	-
nodal marginal zone B-cell lymphoma	odronextamab	Orphan Designation	-
paroxysmal nocturnal hemoglobinuria	pozelimab	Orphan Designation	-
plasma cell myeloma	Lynozyfic	Orphan Designation	-
post-thrombotic syndrome	atoltivimab, odesivimab and maftivimab	Orphan Designation	-
post-traumatic stress disorder	atoltivimab, odesivimab and maftivimab	Orphan Designation	-
post-vaccinal encephalitis	atoltivimab, odesivimab and maftivimab	Orphan Designation	-
primary adult heart tumor	LynozyficDupixent	Orphan Designation	-
protein-losing enteropathy	Veopoz	Orphan Designation	-
psoriasis 14, pustular	rilonacept	Orphan Designation	-
pulmonary arterial hypertension	human anti-PDGF-B monoclonal IgG4 antibody	Orphan Designation	-
retinopathy of prematurity	Eylea	Orphan Designation	-
splenic marginal zone lymphoma	odronextamab	Orphan Designation	-
stage I endometrioid carcinoma	cemiplimab-rwlc	Orphan Designation	-
stage II endometrioid carcinoma	cemiplimab-rwlc	Orphan Designation	-
stage IVb bladder cancer	cemiplimab-rwlc	Orphan Designation	-
sterile multifocal osteomyelitis with periostitis and pustulosis	rilonacept	Orphan Designation	-
systemic-onset juvenile idiopathic arthritis	Interleukin-1 Trap	Orphan Designation	-

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