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Genzyme Corporation — Rare Disease Pipeline | Kisho | Kisho

Genzyme Corporation

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A biopharmaceutical company focused on developing therapies for rare diseases, with 14 FDA-approved drugs and 34 orphan drug designations across 85 conditions, including Fabry disease and neuroacanthocytosis.

Orphan Designations

FDA Approvals

Rare Diseases

News Articles

Disease	Drug(s)	Designation	Approved
Alport syndrome	single stranded, chemically modified oligonucleotide that binds to and inhibits the function of micro RNA-21	Orphan Designation	-
Angelman syndrome	Thyrogen	Orphan Designation	-
B-cell chronic lymphocytic leukemia	Campath	Orphan Designation	-
Bethlem myopathy 1A	Alglucerase injectionCerezyme	Orphan Designation	-
Bicervical bicornuate uterus with patent cervix and vagina	CAPRELSA(r)Campath	Orphan Designation	-
DYRK1A-related intellectual disability syndrome	Mozobil (r)ThyrogenFabrazyme	Orphan Designation	-
Ehlers-Danlos syndrome, kyphoscoliotic type, 2	CAPRELSA(r)	Orphan Designation	-
Fabry disease	VenglustatFabrazyme	Orphan Designation	-
Friedreich ataxia	Mozobil (r)	Orphan Designation	-
Gaucher disease	venglustatRetroviral vector, R-GC and GC gene 1750	Orphan Designation	-
Gaucher disease type I	CERDELGACeredase	Orphan Designation	-
Gaucher disease type II	Alglucerase injectionCerezyme	Orphan Designation	-
Gaucher disease type III	Alglucerase injectionCerezyme	Orphan Designation	-
Gaucher disease-ophthalmoplegia-cardiovascular calcification syndrome	Alglucerase injectionCerezyme	Orphan Designation	-
Kabuki syndrome 1	Campath	Orphan Designation	-
Kaposi's sarcoma	Cerezyme	Orphan Designation	-
Niemann-Pick disease type B	Xenpozyme	Orphan Designation	-
Wolf-Hirschhorn syndrome	Ananain, comosain	Orphan Designation	-
acid sphingomyelinase deficiency	Xenpozyme	Orphan Designation	-
acute flaccid myelitis	anti-thymocyte globulin [rabbit]	Orphan Designation	-
acute lymphoblastic leukemia	anti-thymocyte globulin [rabbit]Clolar	Orphan Designation	-
acute myeloid leukemia	plerixafor	Orphan Designation	-
acute sensory ataxic neuropathy	Thyrogen	Orphan Designation	-
adult-onset non-insulinoma persistent hyperinsulinemic hypoglycemia	CERDELGA	Orphan Designation	-
anosmia for isobutyric acid	Mozobil (r)FabrazymeCeredase	Orphan Designation	-
aortic valve disease 1	Thyrogen	Orphan Designation	-
aortic valve stenosis	Thyrogen	Orphan Designation	-
autism	Qfitlia	Orphan Designation	-
carbon monoxide-induced delayed encephalopathy	CAPRELSA(r)Campath	Orphan Designation	-
cardiomyopathy, familial hypertrophic, 31	Alglucerase injectionCerezyme	Orphan Designation	-
central nervous system mesenchymal non-meningothelial tumor	XenpozymeCerezyme	Orphan Designation	-
central nervous system non-hodgkin lymphoma	Xenpozyme	Orphan Designation	-
chromosome 15q26-qter deletion syndrome	Cystic fibrosis transmembrane conductance regulator	Orphan Designation	-
chronic neurovisceral acid sphingomyelinase deficiency	Xenpozyme	Orphan Designation	-
congenital myopathy 26	Alglucerase injectionCerezyme	Orphan Designation	-
cranioectodermal dysplasia 5	Cystic fibrosis transmembrane conductance regulator	Orphan Designation	-
cystic fibrosis	Lipid/DNA human cystic fibrosis geneCystic fibrosis gene therapy	Orphan Designation	-
dentin dysplasia type I	CERDELGACeredase	Orphan Designation	-
diastrophic dysplasia	Cystic fibrosis transmembrane conductance regulator	Orphan Designation	-
differentiated thyroid carcinoma	Thyrogen	Orphan Designation	-
double nail for fifth toe	Mozobil (r)Ananain, comosainFabrazymeCeredase	Orphan Designation	-
drug rash with eosinophilia and systemic symptoms	Ceredase	Orphan Designation	-
familial hyperaldosteronism type III	Cerezyme	Orphan Designation	-
glycogen storage disease II	1. Myozyme 2. Lumizyme	Orphan Designation	-
graft versus host disease	anti-thymocyte globulin (rabbit)	Orphan Designation	-
hemoglobin--variants for which the chain carrying the mutation 1S unknown or uncertain	Mozobil (r)FabrazymeCeredase	Orphan Designation	-
hemophilia A	Qfitlia	Orphan Designation	-
hemophilia B	Qfitlia	Orphan Designation	-
hypertensive urgency	anti-thymocyte globulin [rabbit]	Orphan Designation	-
idiopathic ductopenia	humanized anti-human CD16 monoclonal antibody	Orphan Designation	-
idiopathic pulmonary fibrosis	human anti-transforming growth factor-B1,2,3	Orphan Designation	-
infantile digital fibromatosis	CAPRELSA(r)	Orphan Designation	-
intellectual developmental disorder, X-linked, syndromic, Pilorge type	Qfitlia	Orphan Designation	-
intellectual disability-severe speech delay-mild dysmorphism syndrome	Qfitlia	Orphan Designation	-
iron poisoning	Deferitrin	Orphan Designation	-
malignant carotid body paraganglioma	Diethylnorspermine (DENSPM)	Orphan Designation	-
malignant central nervous system mesenchymal, non-meningothelial neoplasm	XenpozymeCerezyme	Orphan Designation	-
metastatic melanoma	MART-1 adenoviral gene therapy for malignant melanomaGp100 adenoviral gene therapy	Orphan Designation	-
modifier, X-linked, for Neurofunctional defects	Ceredase	Orphan Designation	-
multisystemic smooth muscle dysfunction syndrome	CAPRELSA(r)	Orphan Designation	-
myelodysplastic syndrome	anti-thymocyte Globulin (rabbit)	Orphan Designation	-
natal teeth-intestinal pseudoobstruction-patent ductus syndrome	CAPRELSA(r)Campath	Orphan Designation	-
neuroacanthocytosis	Mozobil (r)Fabrazyme	Orphan Designation	-
non-central nervous system-localized embryonal carcinoma	XenpozymeCerezyme	Orphan Designation	-
nongerminomatous germ cell tumor	Xenpozyme	Orphan Designation	-
nonimmune chronic idiopathic neutropenia of adults	humanized anti-human CD16 monoclonal antibody	Orphan Designation	-
pediatric acute-onset neuropsychiatric syndrome	Clolar	Orphan Designation	-
pediatric hepatocellular carcinoma	ClolarXenpozymeCerezyme	Orphan Designation	-
pediatric ovarian dysgerminoma	Clolar	Orphan Designation	-
pediatric ovarian germ cell tumor	Clolar	Orphan Designation	-
pediatric-onset Graves disease	Clolar	Orphan Designation	-
peritoneal well differentiated papillary mesothelioma	Thyrogen	Orphan Designation	-
post-treatment Lyme disease syndrome	Diethylnorspermine (DENSPM)	Orphan Designation	-
primary adult heart tumor	CERDELGA	Orphan Designation	-
spatial visualization, aptitude for	Ananain, comosainCeredase	Orphan Designation	-
sporadic adult-onset ataxia of unknown etiology	humanized anti-human CD16 monoclonal antibody	Orphan Designation	-
systemic sclerosis	human anti-transforming growth factor beta 1 monoclonal antibody	Orphan Designation	-
thyroid cancer	Thyrogen	Orphan Designation	-
treatment-refractory schizophrenia	Diethylnorspermine (DENSPM)	Orphan Designation	-
type III hypersensitivity disease	Cerezyme	Orphan Designation	-
tyrosinemia type I	CERDELGACeredase	Orphan Designation	-
well differentiated papillary mesothelioma	Thyrogen	Orphan Designation	-
well-differentiated liposarcoma	Thyrogen	Orphan Designation	-
well-differentiated papillary mesothelial tumour of the pleura	Thyrogen	Orphan Designation	-
xanthinuria type I	CERDELGACeredase	Orphan Designation	-

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