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Pharmacyclics LLC — Rare Disease Pipeline | Kisho | Kisho

Pharmacyclics LLC

Other

Orphan Designations

FDA Approvals

Rare Diseases

News Articles

Disease	Drug(s)	Designation	Approved
B-cell chronic lymphocytic leukemia	IMBRUVICAImbruvicaibrutinib	Orphan Designation	-
Bicervical bicornuate uterus with patent cervix and vagina	Imbruvica	Orphan Designation	-
Char syndrome	Imbruvica	Orphan Designation	-
Gaucher disease type III	Imbruvica	Orphan Designation	-
Meacham syndrome	Imbruvica	Orphan Designation	-
Noonan syndrome 1	Imbruvica	Orphan Designation	-
Sandhoff disease, adult form	Imbruvica	Orphan Designation	-
Waldenstrom macroglobulinemia	Imbruvica	Orphan Designation	-
ataxia and polyneuropathy, adult-onset	Imbruvica	Orphan Designation	-
brain disorder	motexafin gadolinium	Orphan Designation	-
brain injury	motexafin gadolinium	Orphan Designation	-
brain neoplasm	motexafin gadolinium	Orphan Designation	-
carbon monoxide-induced delayed encephalopathy	Imbruvica	Orphan Designation	-
chronic graft versus host disease	Imbruvica	Orphan Designation	-
coccidioidomycosis	IMBRUVICA	Orphan Designation	-
deafness, congenital, and adult-onset progressive leukoencephalopathy	Imbruvica	Orphan Designation	-
diffuse large B-cell lymphoma	ibrutinib	Orphan Designation	-
follicular lymphoma	ibrutinib	Orphan Designation	-
gastric cancer	ibrutinib	Orphan Designation	-
intestinal pseudoobstruction, neuronal, chronic idiopathic, X-linked	IMBRUVICAImbruvica	Orphan Designation	-
malignant pancreatic neoplasm	ibrutinib	Orphan Designation	-
mantle cell lymphoma	Imbruvica	Orphan Designation	-
mitochondrial DNA depletion syndrome 13	IMBRUVICAImbruvica	Orphan Designation	-
multisystemic smooth muscle dysfunction syndrome	Imbruvica	Orphan Designation	-
natal teeth-intestinal pseudoobstruction-patent ductus syndrome	Imbruvica	Orphan Designation	-
necrobiosis lipoidica	IMBRUVICA	Orphan Designation	-
neurodevelopmental disorder with dysmorphic facies and thin corpus callosum	IMBRUVICA	Orphan Designation	-
neuropathy, hereditary sensory and autonomic, adult-onset, with anosmia	Imbruvica	Orphan Designation	-
nodal marginal zone B-cell lymphoma	Imbruvica	Orphan Designation	-
plasma cell myeloma	ibrutinib	Orphan Designation	-
rhizomelic limb shortening with dysmorphic features	IMBRUVICAImbruvica	Orphan Designation	-
splenic marginal zone lymphoma	Imbruvica	Orphan Designation	-

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