Kisho

Vertex Pharmaceuticals Incorporated — Rare Disease Pipeline | Kisho | Kisho

Vertex Pharmaceuticals Incorporated

Other

A biopharmaceutical company focused on developing and commercializing therapies for rare diseases, with 11 FDA-approved drugs and 15 orphan drug designations across 25 conditions.

Orphan Designations

FDA Approvals

Rare Diseases

News Articles

Disease	Drug(s)	Designation	Approved
Dubowitz syndrome	recombinant deriative of C3 transferase	Orphan Designation	-
Fontaine progeroid syndrome	Casgevy	Orphan Designation	-
Ogden syndrome	Casgevy	Orphan Designation	-
Wiedemann-Rautenstrauch syndrome	Casgevy	Orphan Designation	-
acute chest syndrome	Casgevy	Orphan Designation	-
acute disseminated encephalomyelitis	recombinant deriative of C3 transferase	Orphan Designation	-
alveolar capillary dysplasia with misalignment of pulmonary veins	Symdeko	Orphan Designation	-
beta thalassemia	Arginine butyrate	Orphan Designation	-
beta-thalassemia HBB/LCRB	Casgevy	Orphan Designation	-
beta-thalassemia major	Casgevy	-	FDA Approved
brain-lung-thyroid syndrome	SymdekoKalydeco	Orphan Designation	-
bronchiectasis with or without elevated sweat chloride 2	AlyftrekTrikaftaOrkambiKalydeco	Orphan Designation	-
bronchiectasis with or without elevated sweat chloride 3	AlyftrekTrikaftaOrkambiKalydeco	Orphan Designation	-
cystic fibrosis	ALYFTREKTRIKAFTAORKAMBISYMDEKOKALYDECOAlyftrekTrikaftapotassium (benzenesulfonyl)({[6-(3-{2-[1-(trifluoromethyl)cyclopropyl]ethoxy}-1H-pyrazol-1-yl)-2-[(4S)-2,2,4-trimethylpyrrolidin-1-yl]pyridin-3-yl]carbonyl})azanide/tezacaftor/ivacaftorSymdekoD9-ivacaftorOrkambiTezacaftor3-(6-(1-(2,2-difluorobenzo[d] [1,3]dioxyl-5-yl)cyclopropanecarboxamido)-3-methylpyridin-2-yl)benzoic acidKalydeco	Orphan Designation	FDA Approved
cystic fibrosis associated meconium ileus	AlyftrekTrikaftaSymdekoD9-ivacaftorOrkambi	Orphan Designation	-
cystic fibrosis-gastritis-megaloblastic anemia syndrome	AlyftrekTrikaftaSymdekoD9-ivacaftorOrkambiKalydeco	Orphan Designation	-
hereditary persistence of fetal hemoglobin-sickle cell disease syndrome	Casgevy	Orphan Designation	-
lymphedema-distichiasis syndrome	Kalydeco	Orphan Designation	-
membranous glomerulonephritis	povetacicept	Orphan Designation	-
myotonic dystrophy type 1	a phosphorodiamidate morpholino oligomer consisting of 7 repetitive cytosine, adenine, and guanine trimers conjugated via a polyethylene glycol-12 linker to a cyclic peptide and a linear peptide	Orphan Designation	-
neurodevelopmental disorder with intracranial hemorrhage, seizures, and spasticity	Symdeko	Orphan Designation	-
sickle cell disease	CasgevyArginine butyrate	Orphan Designation	FDA Approved
sickle cell-hemoglobin c disease syndrome	Casgevy	Orphan Designation	-
sickle cell-hemoglobin d disease syndrome	Casgevy	Orphan Designation	-
spinal muscular atrophy, distal, autosomal recessive, 6	recombinant deriative of C3 transferase	Orphan Designation	-

← Back to Companies