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Janssen Research & Development, LLC — Rare Disease Pipeline | Kisho | Kisho

Janssen Research & Development, LLC

Other

Orphan Designations

FDA Approvals

Rare Diseases

News Articles

Disease	Drug(s)	Designation	Approved
7q11.23 microduplication syndrome	human anti-integrin receptor avb3/avb5 monoclonal antibody	Orphan Designation	-
AL amyloidosis	Darzalex Faspro	Orphan Designation	-
ALG9-associated autosomal dominant polycystic kidney disease	selective inhibitor of mechanistic target of rapamycin complex 1 (mTORC1)	Orphan Designation	-
Aicardi-Goutieres syndrome	nipocalimab	Orphan Designation	-
B-cell chronic lymphocytic leukemia	cladribine	Orphan Designation	-
Bicervical bicornuate uterus with patent cervix and vagina	Yondelis	Orphan Designation	-
Char syndrome	Yondelis	Orphan Designation	-
Crohn disease	ustekinumab	Orphan Designation	-
DiGeorge syndrome	trabectedin	Orphan Designation	-
Finnish type amyloidosis	human anti-integrin receptor avb3/avb5 monoclonal antibody	Orphan Designation	-
Fontaine progeroid syndrome	macitentan	Orphan Designation	-
Ogden syndrome	Epoetin alpha	Orphan Designation	-
Opitz G/BBB syndrome	trabectedin	Orphan Designation	-
PMM2-congenital disorder of glycosylation	adenovirus associated viral vector serotype 5 containing the RPGR gene	Orphan Designation	-
Sjogren syndrome	SIMPONI ARIA®	Orphan Designation	-
THOC6-related developmental delay-microcephaly-facial dysmorphism syndrome	trabectedin	Orphan Designation	-
active tuberculosis	SIRTURO	Orphan Designation	-
acute myeloid leukemia	bleximenibhumanized monoclonal antibody that targets the a-subunit of the human IL-3 receptor also known as the CD123 antigen; (JNJ-56022473)Cladribine	Orphan Designation	-
adult-onset non-insulinoma persistent hyperinsulinemic hypoglycemia	Darzalex FasproCarvykti	Orphan Designation	-
amyloidosis, hereditary systemic 3	daratumumab	Orphan Designation	-
amyloidosis, hereditary systemic 5	daratumumab	Orphan Designation	-
amyotrophic lateral sclerosis	human umbilical tissue derived cells (hUTC)	Orphan Designation	-
anemia of prematurity	Epoetin alpha	Orphan Designation	-
autoimmune disorder of gastrointestinal tract	SIRTURO	Orphan Designation	-
autoimmune hemolytic anemia	nipocalimab	Orphan Designation	-
autoinflammatory syndrome, familial, Behcet-like 1	SIMPONI ARIA®	Orphan Designation	-
autosomal dominant polycystic kidney disease	selective inhibitor of mechanistic target of rapamycin complex 1 (mTORC1)	Orphan Designation	-
carbon monoxide-induced delayed encephalopathy	Human anti-integrin receptor av monoclonal antibodyYondelis	Orphan Designation	-
chronic inflammatory demyelinating polyradiculoneuropathy	nipocalimab	Orphan Designation	-
chronic relapsing inflammatory optic neuropathy	nipocalimab	Orphan Designation	-
chronic thromboembolic pulmonary hypertension	selexipag	Orphan Designation	-
congenital myasthenic syndrome 3A	Imaavy	Orphan Designation	-
congenital myasthenic syndrome 4C	Imaavy	Orphan Designation	-
congenital myasthenic syndrome 5	Imaavy	Orphan Designation	-
congenital myasthenic syndrome 6	Imaavy	Orphan Designation	-
dengue disease	Arylalkyl 3-ketoindole	Orphan Designation	-
diffuse large B-cell lymphoma	daratumumab	Orphan Designation	-
disease of orbital part of eye adnexa	SIRTURO	Orphan Designation	-
familial visceral amyloidosis	daratumumab	Orphan Designation	-
fetal alcohol syndrome	nipocalimab	Orphan Designation	-
fetal and neonatal alloimmune thrombocytopenia	nipocalimab	Orphan Designation	-
fetal erythroblastosis	nipocalimab	Orphan Designation	-
fetal valproate syndrome	nipocalimab	Orphan Designation	-
filarial elephantiasis	flubendazole	Orphan Designation	-
follicular lymphoma	an autologous Chimeric Antigen Receptors (CAR) T-cell therapy expressing CD19/CD20 bi-specific CARdaratumumab	Orphan Designation	-
frontal lobe epilepsy	macitentan	Orphan Designation	-
hemolytic disease of the newborn with Kell alloimmunization	nipocalimab	Orphan Designation	-
hereditary cryohydrocytosis with reduced stomatin	a synthetic PEGylated thrombopoietin (TPO) mimetic (TPOm) peptide with no sequence homology to endogenous TPO	Orphan Designation	-
hypotonia-failure to thrive-microcephaly syndrome	a synthetic PEGylated thrombopoietin (TPO) mimetic (TPOm) peptide with no sequence homology to endogenous TPO	Orphan Designation	-
immunoglobulin heavy-and-light chain	Darzalex Faspro	Orphan Designation	-
indolent plasma cell myeloma	Carvykti	Orphan Designation	-
inflammatory bowel disease 1	ustekinumab	Orphan Designation	-
inherited retinal dystrophy	adenovirus associated viral vector serotype 5 containing the RPGR gene	Orphan Designation	-
insensitivity to pain with hyperplastic Myelinopathy	a synthetic PEGylated thrombopoietin (TPO) mimetic (TPOm) peptide with no sequence homology to endogenous TPO	Orphan Designation	-
juvenile idiopathic arthritis	sirukumab	Orphan Designation	-
light chain deposition disease	Darzalex Faspro	Orphan Designation	-
lymphatic malformation	flubendazole	Orphan Designation	-
lymphatic system disorder	flubendazole	Orphan Designation	-
malignant neoplasm of abdominal esophagus	SIRTURO	Orphan Designation	-
malignant neoplasm of cervical esophagus	SIRTURO	Orphan Designation	-
malignant neoplasm of thoracic esophagus	SIRTURO	Orphan Designation	-
mantle cell lymphoma	daratumumab	Orphan Designation	-
multisystemic smooth muscle dysfunction syndrome	Human anti-integrin receptor av monoclonal antibodyYondelis	Orphan Designation	-
myasthenia gravis	Imaavy	Orphan Designation	-
myasthenic syndrome, congenital, 1B, fast-channel	Imaavy	Orphan Designation	-
natal teeth-intestinal pseudoobstruction-patent ductus syndrome	Human anti-integrin receptor av monoclonal antibodyYondelis	Orphan Designation	-
neonatal alloimmune neutropenia	nipocalimab	Orphan Designation	-
neurodevelopmental disorder with dysmorphic facies and thin corpus callosum	human anti-integrin receptor avb3/avb5 monoclonal antibody	Orphan Designation	-
neurodevelopmental disorder with or without anomalies of the brain, eye, or heart	human anti-integrin receptor avb3/avb5 monoclonal antibody	Orphan Designation	-
neurodevelopmental-craniofacial syndrome with variable renal and cardiac abnormalities	human anti-integrin receptor avb3/avb5 monoclonal antibody	Orphan Designation	-
non-Hodgkin lymphoma	Cladribine	Orphan Designation	-
non-secretory plasma cell myeloma	Carvykti	Orphan Designation	-
onchocerciasis	flubendazole	Orphan Designation	-
osteopathia striata with cranial sclerosis	human anti-integrin receptor avb3/avb5 monoclonal antibody	Orphan Designation	-
osteosclerotic plasma cell myeloma	Carvykti	Orphan Designation	-
pediatric hepatocellular carcinoma	ustekinumab	Orphan Designation	-
plasma cell myeloma	anti-BCMA x GPRC5D x CD3 trispecific antibodyCarvyktisiltuximab	Orphan Designation	-
polyarticular arthritis	SIMPONI ARIA®	Orphan Designation	-
polyarticular juvenile idiopathic arthritis	sirukumabSIMPONI ARIA®	Orphan Designation	-
polyarticular juvenile rheumatoid arthritis	SIMPONI ARIA®	Orphan Designation	-
polycystic kidney disease 2	selective inhibitor of mechanistic target of rapamycin complex 1 (mTORC1)	Orphan Designation	-
polyneuropathy, inflammatory demyelinating, chronic	nipocalimab	Orphan Designation	-
primary adult heart tumor	Darzalex Faspro	Orphan Designation	-
primary systemic amyloidosis	Darzalex Faspro	Orphan Designation	-
pulmonary non-tuberculous mycobacterial infection	Bedaquiline	Orphan Designation	-
retinal dystrophies primarily involving Bruch's membrane	adenovirus associated viral vector serotype 5 containing the RPGR gene	Orphan Designation	-
retinitis pigmentosa	Human umbilical tissue-derived cells	Orphan Designation	-
rheumatoid factor-positive polyarticular juvenile idiopathic arthritis	sirukumabSIMPONI ARIA®	Orphan Designation	-
sickle cell disease	nipocalimab	Orphan Designation	-
smoldering plasma cell myeloma	Carvykti	Orphan Designation	-
soft tissue sarcoma	Yondelis	Orphan Designation	-
tetrasomy 12p	macitentan	Orphan Designation	-
trichothiodystrophy	Epoetin alpha	Orphan Designation	-
twin to twin transfusion syndrome	a synthetic PEGylated thrombopoietin (TPO) mimetic (TPOm) peptide with no sequence homology to endogenous TPO	Orphan Designation	-

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