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Gilead Sciences, Inc. — Rare Disease Pipeline | Kisho | Kisho

Gilead Sciences, Inc.

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A biopharmaceutical company focused on developing therapies for rare diseases, with 42 orphan drug designations and active involvement in 89 rare conditions, including mitochondrial DNA depletion syndrome.

Orphan Designations

FDA Approvals

Rare Diseases

News Articles

Disease	Drug(s)	Designation	Approved
B-cell chronic lymphocytic leukemia	6-Amino-9-[(3R)-1-(but-2-ynoyl)pyrrolidin-3-yl]-7-(4-phenoxyphenyl)-7,9-dihydro-8H-purin-8-one monohydrochlorideZydeligidelalisib	Orphan Designation	-
COVID-19	remdesivir	Orphan Designation	-
COVID-19–associated multisystem inflammatory syndrome in children	remdesivir	Orphan Designation	-
Crohn disease	filgotinib maleate	Orphan Designation	-
Ebola hemorrhagic fever	Remdesivir	Orphan Designation	-
HIV infectious disease	Tybost®Biktarvy	Orphan Designation	-
Kabuki syndrome 1	Descovy	Orphan Designation	-
Kaposi's sarcoma	Tybost®Biktarvy	Orphan Designation	-
MALT lymphoma	idelalisib	Orphan Designation	-
NPHP3-related Meckel-like syndrome	Zydelig	Orphan Designation	-
Singleton-Merten syndrome 1	Tybost®Descovy	Orphan Designation	-
Sjogren syndrome	Harvoni	Orphan Designation	-
THOC6-related developmental delay-microcephaly-facial dysmorphism syndrome	Zydelig	Orphan Designation	-
Waldenstrom macroglobulinemia	idelalisib	Orphan Designation	-
Weaver syndrome	Zydelig	Orphan Designation	-
abetalipoproteinemia	2-[4-[[(2R)-2-ethoxy-3-[4-(trifluoromethyl)phenoxy]propyl]thio]-2-methylphenoxy]acetic acid (1:1) lysine dihydrate	Orphan Designation	-
acute myeloid leukemia	humanized monoclonal antibody of the IgG4 kappa isotype targeting CD47	Orphan Designation	-
acute myeloid leukemia with minimal differentiation	humanized monoclonal antibody of the IgG4 kappa isotype targeting CD47	Orphan Designation	-
alcohol dependence	TRUVADA	-	FDA Approved
bacterial pneumonia	aztreonam	Orphan Designation	-
baritosis	Cayston	Orphan Designation	-
carbon monoxide-induced delayed encephalopathy	2-[4-[[(2-R)-2-ethoxy-3-[4-(trifluoromethyl)phenoxy]propyl]thio]-2-methylphenoxy]acetic acid (1:1) lysine dihydrate	Orphan Designation	-
childhood acute myeloid leukemia	humanized monoclonal antibody of the IgG4 kappa isotype targeting CD47	Orphan Designation	-
chronic graft versus host disease	Sovaldi	Orphan Designation	-
chronic hepatitis B virus infection	Harvoni	Orphan Designation	-
chronic hepatitis C virus infection	Harvoni	Orphan Designation	-
chronic inflammatory demyelinating polyradiculoneuropathy	Sovaldi	Orphan Designation	-
chronic primary adrenal insufficiency	Sovaldi	Orphan Designation	-
chronic recurrent multifocal osteomyelitis	Sovaldi	Orphan Designation	-
cleidocranial dysplasia 1	Tybost®Descovy	Orphan Designation	-
congenital enterovirus infection	tenofovir disoproxil fumarate	Orphan Designation	-
congenital human immunodeficiency virus	Tybost®Biktarvy	Orphan Designation	-
congenital syphilis	tenofovir disoproxil fumarate	Orphan Designation	-
extranodal nasal NK/T cell lymphoma	idelalisib	Orphan Designation	-
familial long QT syndrome	4-(pyrimidin-2-ylmethyl)-7-[4-(trifluoromethoxy)phenyl]-3,4-dihydro-1,4-benzoxazepin-5(2H)-one	Orphan Designation	-
fetal cytomegalovirus syndrome	tenofovir disoproxil fumarate	Orphan Designation	-
follicular lymphoma	Zydelig	Orphan Designation	-
gastric cancer	recombinant humanized anti-matrix metalloprotein 9 (MMP9) monoclonal antibody IgG4	Orphan Designation	-
gram-negative bacterial infections	aztreonam	Orphan Designation	-
hepatitis B virus infection	Harvoni	Orphan Designation	-
hepatitis C virus infection	Harvoni	Orphan Designation	-
hepatitis D virus infection	bulevirtide	Orphan Designation	-
homozygous 11P15-p14 deletion syndrome	2-[4-[[(2R)-2-ethoxy-3-[4-(trifluoromethyl)phenoxy]propyl]thio]-2-methylphenoxy]acetic acid (1:1) lysine dihydrate	Orphan Designation	-
homozygous familial hypercholesterolemia	2-[4-[[(2R)-2-ethoxy-3-[4-(trifluoromethyl)phenoxy]propyl]thio]-2-methylphenoxy]acetic acid (1:1) lysine dihydrate	Orphan Designation	-
idiopathic pulmonary fibrosis	Recombinant humanized anti-LOXL2 monoclonal antibodyambrisentan	Orphan Designation	-
inflammatory bowel disease 1	filgotinib maleate	Orphan Designation	-
inhalational anthrax	Cayston	Orphan Designation	-
inhalational botulism	Cayston	Orphan Designation	-
intellectual disability, autosomal dominant 1	Tybost®Descovy	Orphan Designation	-
lymphoplasmacytic lymphoma	idelalisib	Orphan Designation	-
lymphoplasmacytic lymphoma without IgM production	idelalisib	Orphan Designation	-
malignant carotid body paraganglioma	cicletanine hydrochloride	Orphan Designation	-
malignant glioma	sacituzumab govitecan (SG)	Orphan Designation	-
malignant pancreatic neoplasm	sacituzumab govitecanrecombinant humanized anti-LOXL2 monoclonal antibody, IgG4(S241P)	Orphan Designation	-
meconium aspiration syndrome	Cayston	Orphan Designation	-
mitochondrial DNA depletion syndrome 13	Zydelig	Orphan Designation	-
multiple congenital anomalies-hypotonia-seizures syndrome 2	Viread	Orphan Designation	-
multisystem inflammatory syndrome in children and adults	Epclusa	Orphan Designation	-
multisystemic smooth muscle dysfunction syndrome	2-[4-[[(2-R)-2-ethoxy-3-[4-(trifluoromethyl)phenoxy]propyl]thio]-2-methylphenoxy]acetic acid (1:1) lysine dihydrateZydelig	Orphan Designation	-
mycosis fungoides	idelalisib	Orphan Designation	-
myelodysplastic syndrome	Magrolimab	Orphan Designation	-
myelofibrosis	Recombinant humanized anti-LOXL2 monoclonal antibody, IgG4(S241P)	Orphan Designation	-
natal teeth-intestinal pseudoobstruction-patent ductus syndrome	2-[4-[[(2-R)-2-ethoxy-3-[4-(trifluoromethyl)phenoxy]propyl]thio]-2-methylphenoxy]acetic acid (1:1) lysine dihydrate	Orphan Designation	-
neonatal encephalomyopathy-cardiomyopathy-respiratory distress syndrome	Zydelig	Orphan Designation	-
neurodevelopmental disorder with or without anomalies of the brain, eye, or heart	Zydelig	Orphan Designation	-
neurodevelopmental-craniofacial syndrome with variable renal and cardiac abnormalities	Zydelig	Orphan Designation	-
nodal marginal zone B-cell lymphoma	idelalisib	Orphan Designation	-
occupational lung disease	Cayston	Orphan Designation	-
pediatric acute-onset neuropsychiatric syndrome	EpclusaSovaldiHarvoniViread	Orphan Designation	-
pediatric hepatocellular carcinoma	filgotinib maleateEpclusaSovaldiHarvoniViread	Orphan Designation	-
pediatric ovarian dysgerminoma	EpclusaSovaldiHarvoniViread	Orphan Designation	-
pediatric ovarian germ cell tumor	Viread	Orphan Designation	-
pediatric-onset Graves disease	filgotinib maleateEpclusaSovaldiHarvoniViread	Orphan Designation	-
post 5-alpha-reductase inhibitors treatment syndrome	5-(4-Cyclopropyl-1H-imidazol-l-yl)-2-fluoro-N-(6-(4-isopropyl-4H-1,2,4-triazol-3-yl)pyridin-2-yl)-4-methylbenzamide	Orphan Designation	-
post-treatment Lyme disease syndrome	5-(4-Cyclopropyl-1H-imidazol-l-yl)-2-fluoro-N-(6-(4-isopropyl-4H-1,2,4-triazol-3-yl)pyridin-2-yl)-4-methylbenzamidecicletanine hydrochloride	Orphan Designation	-
primary biliary cholangitis	Livdelzi	Orphan Designation	-
primary biliary cholangitis 1	Livdelzi	Orphan Designation	-
primary biliary cholangitis 2	Livdelzi	Orphan Designation	-
primary biliary cholangitis 4	Livdelzi	Orphan Designation	-
primary biliary cholangitis/primary sclerosing cholangitis and autoimmune hepatitis overlap syndrome	Livdelzi	Orphan Designation	-
primary sclerosing cholangitis	simtuzumab	Orphan Designation	-
pulmonary arterial hypertension	ambrisentan/tadalafil fixed combination	Orphan Designation	-
sclerosing cholangitis	2-[3-(2-chloro-4-{[5-cyclopropyl-3-(2,6-dichlorophenyl)-1,2-oxazol-4-yl]methoxy}phenyl)-3-hydroxyazetidin-1-yl]pyridine-4-carboxylic acid¿2-amino-2-(hydroxymethyl)propane-1,3-diol (1/1)	Orphan Designation	-
severe acute respiratory syndrome	remdesivir	Orphan Designation	-
small cell lung carcinoma	sacituzumab govitecan	Orphan Designation	-
spinal muscular atrophy, type III	EpclusaSovaldi	Orphan Designation	-
splenic marginal zone lymphoma	idelalisib	Orphan Designation	-
treatment-refractory schizophrenia	5-(4-Cyclopropyl-1H-imidazol-l-yl)-2-fluoro-N-(6-(4-isopropyl-4H-1,2,4-triazol-3-yl)pyridin-2-yl)-4-methylbenzamidecicletanine hydrochloride	Orphan Designation	-
twin to twin transfusion syndrome	Cayston	Orphan Designation	-

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