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Chiesi USA, Inc. — Rare Disease Pipeline | Kisho | Kisho

Chiesi USA, Inc.

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This company focuses on developing treatments for rare diseases, with 11 FDA-approved drugs and 14 orphan drug designations across 40 active rare disease indications.

Orphan Designations

FDA Approvals

Rare Diseases

News Articles

Disease	Drug(s)	Designation	Approved
African iron overload	Ferriprox	Orphan Designation	-
Diamond-Blackfan anemia 12	Revcovi	Orphan Designation	-
Friedreich ataxia	deferiprone	Orphan Designation	-
Krabbe disease	recombinant human galactocerebrosidase (rhGALC);	Orphan Designation	-
acromegaly	MYCAPSSA (formerly Octreolin)	Orphan Designation	-
acute flaccid myelitis	recombinant human porphobilinogen deaminaserecombinant human porphobilinogen deaminase, erythropoetic form	Orphan Designation	-
acute intermittent porphyria	recombinant human porphobilinogen deaminaserecombinant human porphobilinogen deaminase, erythropoetic form	Orphan Designation	-
adenosine monophosphate deaminase deficiency	Revcovi	Orphan Designation	-
alpha-mannosidosis	Lamzede	Orphan Designation	-
anemia, hypochromic microcytic with iron overload	Ferriprox	Orphan Designation	-
carcinoid syndrome	Octreotide (acetate)	Orphan Designation	-
central nervous system mesenchymal non-meningothelial tumor	Lamzede	Orphan Designation	-
central nervous system non-hodgkin lymphoma	Lamzede	Orphan Designation	-
cholestasis, progressive familial intrahepatic, 6	Ferriprox	Orphan Designation	-
congenital lethal erythroderma	Curosurf	Orphan Designation	-
deficiency of adenosine deaminase 2	Revcovi	Orphan Designation	-
hemoglobinopathy	recombinant human galactocerebrosidase (rhGALC);	Orphan Designation	-
major depressive disorder	Curosurf	Orphan Designation	-
malignant central nervous system mesenchymal, non-meningothelial neoplasm	Lamzede	Orphan Designation	-
microcytic anemia with liver iron overload	Ferriprox	Orphan Designation	-
neonatal encephalomyopathy-cardiomyopathy-respiratory distress syndrome	synthetic surfactant comprised of DPPC, POPG Na, synthetic SP-C analogue and synthetic SP-B analogue	Orphan Designation	-
neurodegeneration with brain iron accumulation	deferiprone	Orphan Designation	-
neurodegeneration with brain iron accumulation 7	deferiprone	Orphan Designation	-
neurodegeneration with brain iron accumulation 8	deferiprone	Orphan Designation	-
non-central nervous system-localized embryonal carcinoma	Lamzede	Orphan Designation	-
nongerminomatous germ cell tumor	Lamzede	Orphan Designation	-
pediatric hepatocellular carcinoma	Lamzede	Orphan Designation	-
pediatric ovarian dysgerminoma	Lamzede	Orphan Designation	-
pediatric-onset Graves disease	Lamzede	Orphan Designation	-
porphyria, acute intermittent, nonerythroid variant	recombinant human porphobilinogen deaminaserecombinant human porphobilinogen deaminase, erythropoetic form	Orphan Designation	-
post 5-alpha-reductase inhibitors treatment syndrome	Curosurf	Orphan Designation	-
post-treatment Lyme disease syndrome	Curosurf	Orphan Designation	-
preterm premature rupture of the membranes	synthetic surfactant comprised of DPPC, POPG Na, synthetic SP-C analogue and synthetic SP-B analogue	Orphan Designation	-
respiratory distress syndrome in premature infants	Curosurf	Orphan Designation	-
serum sickness	Ferriprox	Orphan Designation	-
sickle cell disease	recombinant human galactocerebrosidase (rhGALC);	Orphan Designation	-
superficial siderosis	deferiprone	Orphan Designation	-
systemic sclerosis	monomethoxypolyethylene glycol recombinant adenosine deaminase	Orphan Designation	-
treatment-refractory schizophrenia	Curosurf	Orphan Designation	-
twin to twin transfusion syndrome	Ferriprox	Orphan Designation	-

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