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Ferring Pharmaceuticals Inc. — Rare Disease Pipeline | Kisho | Kisho

Ferring Pharmaceuticals Inc.

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Specializing in therapies for rare diseases, the company has 9 FDA-approved drugs and 7 orphan drug designations, actively addressing 39 conditions, including various forms of primary ovarian failure and mild hemophilia A.

Orphan Designations

FDA Approvals

Rare Diseases

News Articles

Disease	Drug(s)	Designation	Approved
Bicervical bicornuate uterus with patent cervix and vagina	Desmopressin acetate	Orphan Designation	-
Bloom syndrome	Lutrepulse	Orphan Designation	-
Char syndrome	Desmopressin acetate	Orphan Designation	-
DYRK1A-related intellectual disability syndrome	Acthrel	Orphan Designation	-
Rubinstein-Taybi syndrome due to CREBBP mutations	Lutrepulse	Orphan Designation	-
acquired primary ovarian failure	MENOPUR	-	FDA Approved
anosmia for isobutyric acid	Acthrel	Orphan Designation	-
autoimmune primary ovarian failure	MENOPUR	-	FDA Approved
carbon monoxide-induced delayed encephalopathy	Desmopressin acetate	Orphan Designation	-
double nail for fifth toe	Acthrel	Orphan Designation	-
ectopic ACTH secretion syndrome	Acthrel	Orphan Designation	-
ectopic Cushing syndrome	Somatostatin	Orphan Designation	-
ectopic hormone secretion syndrome associated with neoplasia	Acthrel	Orphan Designation	-
ectopic pregnancy	Acthrel	Orphan Designation	-
ectopic thymus	Acthrel	Orphan Designation	-
encephalopathy, recurrent, of childhood	Rebyota	Orphan Designation	-
esophageal varices	Terlipressin	Orphan Designation	-
hypotonia-failure to thrive-microcephaly syndrome	Acthrel	Orphan Designation	-
ileum cancer	Somatostatin	Orphan Designation	-
inherited primary ovarian failure	MENOPUR	-	FDA Approved
insensitivity to pain with hyperplastic Myelinopathy	Acthrel	Orphan Designation	-
mild hemophilia A	Desmopressin acetate	Orphan Designation	-
multiple congenital anomalies-hypotonia-seizures syndrome 2	Lutrepulse	Orphan Designation	-
multisystemic smooth muscle dysfunction syndrome	Desmopressin acetate	Orphan Designation	-
natal teeth-intestinal pseudoobstruction-patent ductus syndrome	Desmopressin acetate	Orphan Designation	-
neuroacanthocytosis	Acthrel	Orphan Designation	-
non-secreting chemodectoma	Somatostatin	Orphan Designation	-
non-secreting paraganglioma	Somatostatin	Orphan Designation	-
ovarian ectopic pregnancy	Acthrel	Orphan Designation	-
primary ovarian failure	MENOPUR	-	FDA Approved
recurrent acute pancreatitis	Rebyota	Orphan Designation	-
recurrent hypersomnia	Rebyota	Orphan Designation	-
recurrent idiopathic neuroretinitis	Rebyota	Orphan Designation	-
recurrent metabolic encephalomyopathic crises-rhabdomyolysis-cardiac arrhythmia-intellectual disability syndrome	Rebyota	Orphan Designation	-
recurrent respiratory papillomatosis	Rebyota	Orphan Designation	-
sporadic secreting paraganglioma	Somatostatin	Orphan Designation	-
twin to twin transfusion syndrome	Acthrel	Orphan Designation	-
variegate porphyria	Somatostatin	Orphan Designation	-
von Willebrand disease (hereditary or acquired)	Desmopressin acetate	Orphan Designation	-

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