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GlaxoSmithKline LLC — Rare Disease Pipeline | Kisho | Kisho

GlaxoSmithKline LLC

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A biopharmaceutical company engaged in developing treatments for rare diseases, with 31 FDA-approved drugs and 16 orphan drug designations across 63 conditions, including essential thrombocythemia and epilepsy.

Orphan Designations

FDA Approvals

Rare Diseases

News Articles

Disease	Drug(s)	Designation	Approved
8p inverted duplication/deletion syndrome	Atovaquone	Orphan Designation	-
Adams-Oliver syndrome 1	Dehydroepiandrosterone	Orphan Designation	-
Au-Kline syndrome	Atovaquone	Orphan Designation	-
B-cell non-Hodgkin lymphoma	Bexxar	Orphan Designation	-
Bicervical bicornuate uterus with patent cervix and vagina	Bexxar	Orphan Designation	-
Char syndrome	Bexxar	Orphan Designation	-
Diamond-Blackfan anemia 11	Dehydroepiandrosterone	Orphan Designation	-
Gaucher disease type III	NUCALA®	Orphan Designation	-
MOGS-congenital disorder of glycosylation	Atovaquone	Orphan Designation	-
Plasmodium falciparum malaria	MALARONE	-	FDA Approved
Sandhoff disease, adult form	NUCALA®	Orphan Designation	-
adrenal cortex carcinoma	Atovaquone	Orphan Designation	-
adult acute respiratory distress syndrome	Colfosceril palmitate, cetyl alcohol, tyloxapol	Orphan Designation	-
adult neuronal ceroid lipofuscinosis	NUCALAZejula	Orphan Designation	-
adult pleomorphic rhabdomyosarcoma	NUCALAZejula	Orphan Designation	-
adult-onset non-insulinoma persistent hyperinsulinemic hypoglycemia	NUCALAZejula	Orphan Designation	-
adult-onset proximal spinal muscular atrophy, autosomal dominant	NUCALAZejula	Orphan Designation	-
allergic asthma	ADVAIR DISKUS	-	FDA Approved
amyotrophic lateral sclerosis	anti-nogo-A monoclonal antibody	Orphan Designation	-
anuria	Atovaquone	Orphan Designation	-
asthma	ADVAIR DISKUS	-	FDA Approved
ataxia and polyneuropathy, adult-onset	NUCALA®	Orphan Designation	-
atypical Werner syndrome	Flolan	Orphan Designation	-
autosomal recessive osteopetrosis 3	Flolan	Orphan Designation	-
bipolar I disorder	LAMICTAL	-	FDA Approved
carbon monoxide-induced delayed encephalopathy	Bexxar	Orphan Designation	-
central nervous system lupus	Dehydroepiandrosterone	Orphan Designation	-
childhood-onset nemaline myopathy	otelixizumab	Orphan Designation	-
chronic obstructive pulmonary disease	INCRUSE ELLIPTABREO ELLIPTARELENZA	-	FDA Approved
congenital hypotonia, epilepsy, developmental delay, and digital anomalies	Atovaquone	Orphan Designation	-
cough variant asthma	ADVAIR DISKUS	-	FDA Approved
deafness, congenital, and adult-onset progressive leukoencephalopathy	NUCALA®	Orphan Designation	-
discrimination, Two-point, reduction 1N	Dehydroepiandrosterone	Orphan Designation	-
ectopic Cushing syndrome	Flolan	Orphan Designation	-
eosinophilic granulomatosis with polyangiitis	NUCALA	Orphan Designation	-
epilepsy	LAMICTAL	-	FDA Approved
essential thrombocythemia	OJJAARA	-	FDA Approved
familial pulmonary arterial hypertension leucopenia and atrial septal defect	Flolan	Orphan Designation	-
heritable pulmonary arterial hypertension	Flolan	Orphan Designation	-
herpes simplex virus keratitis	acyclovir	Orphan Designation	-
hypereosinophilic syndrome	NUCALA®	Orphan Designation	-
idiopathic pulmonary arterial hypertension	Flolan	Orphan Designation	-
intrinsic asthma	ADVAIR DISKUS	-	FDA Approved
lethal congenital glycogen storage disease of heart	Atovaquone	Orphan Designation	-
malaria	MALARONE	-	FDA Approved
multisystemic smooth muscle dysfunction syndrome	Bexxar	Orphan Designation	-
myelofibrosis	Ojjaara	Orphan Designation	-
natal teeth-intestinal pseudoobstruction-patent ductus syndrome	Bexxar	Orphan Designation	-
nemaline myopathy 2	otelixizumab	Orphan Designation	-
neuropathy, hereditary sensory and autonomic, adult-onset, with anosmia	NUCALA®	Orphan Designation	-
new-onset refractory status epilepticus	otelixizumab	Orphan Designation	-
ovarian cancer	Zejula	Orphan Designation	-
pediatric systemic lupus erythematosus	Dehydroepiandrosterone	Orphan Designation	-
polycythemia	OJJAARA	-	FDA Approved
primary adult heart tumor	NUCALAZejula	Orphan Designation	-
primary biliary cholangitis	linerixibat	Orphan Designation	-
pulmonary arterial hypertension	Flolan	Orphan Designation	-
renal cell carcinoma	Iboctadekin	Orphan Designation	-
secondary pulmonary alveolar proteinosis	Flolan	Orphan Designation	-
secondary pulmonary hemosiderosis	Flolan	Orphan Designation	-
small cell lung carcinoma	risvutatug rezetecan	Orphan Designation	-
status asthmaticus	ADVAIR DISKUS	-	FDA Approved
systemic lupus erythematosus	Dehydroepiandrosterone	Orphan Designation	-

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