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Novartis Pharmaceuticals Corporation — Rare Disease Pipeline | Kisho | Kisho

Novartis Pharmaceuticals Corporation

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A global biopharmaceutical company focused on developing treatments for rare diseases, with 109 orphan drug designations and active involvement in 271 rare conditions.

109

Orphan Designations

FDA Approvals

271

Rare Diseases

News Articles

Disease	Drug(s)	Designation	Approved
5-nucleotidase syndrome	IgG-like bispecific monoclonal antibody (mAb) composed of the anti-IL-1beta mAb canakinumab and the anti-IL-18 mAb CMK389	Orphan Designation	-
ALG9-associated autosomal dominant polycystic kidney disease	a chemically modified full phosphorothioate-backbone anti-miR-17 oligonucleotide	Orphan Designation	-
African iron overload	Exjade; Jadenu Sprinkles	Orphan Designation	-
B-cell chronic lymphocytic leukemia	TisagenlecleucelARZERRAnelarabine	Orphan Designation	-
B-lymphoblastic leukemia/lymphoma with t(9;22)(q34.1;q11.2)	Gleevec	Orphan Designation	-
Bicervical bicornuate uterus with patent cervix and vagina	Tafinlar(r) Capsules a nd Mekinist(r) TabletsMekinistdabrafenibArranonMulti-ligand somatostatin analogGleevec	Orphan Designation	-
CINCA syndrome	Ilaris	Orphan Designation	-
Char syndrome	Tafinlar(r) Capsules a nd Mekinist(r) TabletsMekinistpasireotideGleevec	Orphan Designation	-
Charcot-Marie-Tooth disease type 1A	Double-stranded small-interfering ribonucleic acid (siRNA) comprised of an antisense strand complementary to a targeted sequence within human PMP22 messenger RNA and a nucleotide sense strand linked to a fatty acid motif	Orphan Designation	-
Chediak-Higashi syndrome	selective deacylglycerol acyltransferase 1 inhibitor	Orphan Designation	-
Cushing disease due to pituitary adenoma	Signifor	Orphan Designation	-
DiGeorge syndrome	Arranon	Orphan Designation	-
Down syndrome	Ursodiol	Orphan Designation	-
Dubowitz syndrome	anti human Nogo-A human monoclonal antibody	Orphan Designation	-
Ehlers-Danlos syndrome, spondylodysplastic type, 2	Votrient	Orphan Designation	-
FG syndrome 1	Coartem	Orphan Designation	-
Finnish type amyloidosis	spartalizumab	Orphan Designation	-
GM1 gangliosidosis	Multi-ligand somatostatin analog	Orphan Designation	-
GRACILE syndrome	Exjade; Jadenu Sprinkles	Orphan Designation	-
Gaucher disease type III	Vijoice	Orphan Designation	-
Gorham-Stout disease	Sandostatin LAR	Orphan Designation	-
HER2 positive breast carcinoma	lapatinib ditosylate hydrochloridelapatinib	Orphan Designation	-
Hodgkin's lymphoma, lymphocytic depletion	Simulect	Orphan Designation	-
Hodgkins lymphoma	MEKINIST and TAFINLARMEKINISTpanobinostat	Orphan Designation	-
Houge-Janssens syndrome 4	Guanethidine monosulfate	Orphan Designation	-
Huntington disease	votoplamBranaplam	Orphan Designation	-
Huntington disease-like 1	Branaplam	Orphan Designation	-
Huntington disease-like 2	votoplamBranaplam	Orphan Designation	-
Kabuki syndrome 1	Kymriah (tisagenlecleucel)PromactaArranon	Orphan Designation	-
Khan-Khan-Katsanis syndrome	Tafinlar(r) Capsules a nd Mekinist(r) Tablets	Orphan Designation	-
Kleefstra syndrome 1	ZYKADIA	Orphan Designation	-
Li-Fraumeni syndrome	Votrient	Orphan Designation	-
Loeys-Dietz syndrome	Ilaris	Orphan Designation	-
Loeys-Dietz syndrome 1	Coartem	Orphan Designation	-
Matthew-Wood syndrome	Ursodiol	Orphan Designation	-
Mowat-Wilson syndrome	Promacta	Orphan Designation	-
Muckle-Wells syndrome	Ilaris	Orphan Designation	-
Noonan syndrome with multiple lentigines	Sandostatin LAR	Orphan Designation	-
Noonan syndrome-like disorder with loose anagen hair 2	Gleevec	Orphan Designation	-
Oncogenic osteomalacia	Zometa, Zabel	Orphan Designation	-
PIK3CA-related overgrowth spectrum	Vijoice	Orphan Designation	-
Philadelphia-positive myelogenous leukemia	Gleevec	Orphan Designation	-
Roberts-SC phocomelia syndrome	Promacta	Orphan Designation	-
Sandhoff disease, adult form	Vijoice	Orphan Designation	-
Sjogren syndrome	ILARIS	Orphan Designation	-
Smouldering systemic mastocytosis	Gleevec	Orphan Designation	-
T-cell adult acute lymphocytic leukemia	Arranon	Orphan Designation	-
T-cell childhood lymphoblastic lymphoma	Arranon	Orphan Designation	-
T-lymphoblastic lymphoma	Arranon	Orphan Designation	-
TNF receptor 1-associated periodic fever syndrome	Ilaris	Orphan Designation	-
Takayasu arteritis	Amphotericin B inhalation powder	Orphan Designation	-
Tn polyagglutination syndrome	Ilaris	Orphan Designation	-
Uner Tan Syndrome	ILARIS	Orphan Designation	-
Waldenstrom macroglobulinemia	everolimus	Orphan Designation	-
Weaver syndrome	ZYKADIA	Orphan Designation	-
Westphal disease	votoplam	Orphan Designation	-
Woodhouse-Sakati syndrome	patupilone	Orphan Designation	-
Yunis-Varon syndrome	Tafinlar(r) Capsules a nd Mekinist(r) TabletsPromactaZYKADIA	Orphan Designation	-
abetalipoproteinemia	inclisiran	Orphan Designation	-
absence of the pulmonary artery	Tobramycin	Orphan Designation	-
acromegaly	pasireotideSandostatin LAR	Orphan Designation	-
active cochlear Meniere disease	canakinumab	Orphan Designation	-
active cochleovestibular Meniere disease	canakinumab	Orphan Designation	-
active peptic ulcer disease	canakinumab	Orphan Designation	-
active tuberculosis	canakinumabclofazimine	Orphan Designation	-
active vestibular Meniere disease	canakinumab	Orphan Designation	-
acute disseminated encephalomyelitis	anti human Nogo-A human monoclonal antibody	Orphan Designation	-
acute lung injury	Ilaris	Orphan Designation	-
acute lymphoblastic leukemia	KymriahKymriah (tisagenlecleucel)Arranon	Orphan Designation	FDA Approved
acute myeloid leukemia	Rydapt	Orphan Designation	-
adult Refsum disease	Afinitor	Orphan Designation	-
adult embryonal tumor with multilayered rosettes, c19mc-altered	Fabhalta	Orphan Designation	-
adult neuronal ceroid lipofuscinosis	KymriahScemblixRydaptSigniforGleevec	Orphan Designation	-
adult pleomorphic rhabdomyosarcoma	KymriahScemblixRydaptSigniforGleevec	Orphan Designation	-
adult-onset Still disease	canakinumab	Orphan Designation	-
adult-onset chronic progressive external ophthalmoplegia with mitochondrial myopathy	Fabhalta	Orphan Designation	-
adult-onset non-insulinoma persistent hyperinsulinemic hypoglycemia	KymriahcapmatinibScemblixRydaptSigniforGleevec	Orphan Designation	-
adult-onset proximal spinal muscular atrophy, autosomal dominant	KymriahScemblixRydaptSigniforGleevec	Orphan Designation	-
aggressive systemic mastocytosis	Gleevec	Orphan Designation	-
alveolar soft part sarcoma	Votrient	Orphan Designation	-
amyotrophic lateral sclerosis	sotuletinib	Orphan Designation	-
antisynthetase syndrome	IgG-like bispecific monoclonal antibody (mAb) composed of the anti-IL-1beta mAb canakinumab and the anti-IL-18 mAb CMK389	Orphan Designation	-
aplastic anemia	Promacta	Orphan Designation	-
ataxia and polyneuropathy, adult-onset	Vijoice	Orphan Designation	-
attention deficit-hyperactivity disorder	RITALIN	-	FDA Approved
autism spectrum disorder 1	Tafinlar(r) Capsules a nd Mekinist(r) Tablets	Orphan Designation	-
autoimmune thrombocytopenic purpura	Promacta	Orphan Designation	-
autosomal dominant polycystic kidney disease	a chemically modified full phosphorothioate-backbone anti-miR-17 oligonucleotide	Orphan Designation	-
autosomal dominant progressive nephropathy with hypertension	Afinitor	Orphan Designation	-
autosomal recessive familial Mediterranean fever	ILARIS	Orphan Designation	-
brain glioma	MekinistTafinlar	Orphan Designation	-
breast carcinoma by gene expression profile	Sandostatin LAR	Orphan Designation	-
bronchiectasis	Tobramycin	Orphan Designation	-
candidiasis, invasive	recombinant human monoclonal antibody to hsp90	Orphan Designation	-
carbon monoxide-induced delayed encephalopathy	Tafinlar(r) Capsules a nd Mekinist(r) TabletsMekinistdabrafenibKymriah (tisagenlecleucel)MEKINIST and TAFINLARTAFINLARMEKINISTpasireotideArranonMulti-ligand somatostatin analogGleevec	Orphan Designation	-
cataract 13 with adult I phenotype	Fabhalta	Orphan Designation	-
cerebellar ataxia	N-(4,4-difluorocyclohexyl)-2-(3-methyl-1H-pyrazol-1-yl)-6-morpholinopyrimidin-4-amine	Orphan Designation	-
cerebellar ataxia with neuropathy and bilateral vestibular areflexia syndrome	ILARIS	Orphan Designation	-
cervical cancer	HYCAMTIN	-	FDA Approved
chromosome 18q deletion syndrome	Coartem	Orphan Designation	-
chronic myelogenous leukemia, BCR-ABL1 positive	ScemblixTasignaGleevec	Orphan Designation	-
coccidioidomycosis	ILARIS	Orphan Designation	-
congenital	human IgG1 anti-human cytomegalovirus monoclonal antibodies LJP538 and LJP539	Orphan Designation	-
congenital anomalies of kidney and urinary tract 3	Kymriah (tisagenlecleucel)	Orphan Designation	-
congenital heart disease	human IgG1 anti-human cytomegalovirus monoclonal antibodies LJP538 and LJP539	Orphan Designation	-
congenital lethal erythroderma	TAFINLAR	Orphan Designation	-
congenital microcephaly - severe encephalopathy - progressive cerebral atrophy syndrome	Guanethidine monosulfate	Orphan Designation	-
congenital tracheomalacia	Tobramycin	Orphan Designation	-
craniofacial-deafness-hand syndrome	Sandostatin LAR	Orphan Designation	-
craniosynostosis, Philadelphia type	Gleevec	Orphan Designation	-
cryopyrin-associated periodic syndrome	Ilaris	Orphan Designation	-
cutaneous mastocytoma	Tafinlar(r) Capsules a nd Mekinist(r) Tablets	Orphan Designation	-
cutaneous neuroendocrine carcinoma	humanized monoclonal antibody against human PD-1octreotideAfinitor	Orphan Designation	-
cutaneous photosensitivity-lethal colitis syndrome	Sandostatin LAR	Orphan Designation	-
cystinosis	autologous CD34+ enriched cells transduced with a lentiviral vector containing RNA resulting in codon-optimized cDNA encoding for functional human cystinosin	Orphan Designation	-
deafness, congenital, and adult-onset progressive leukoencephalopathy	Vijoice	Orphan Designation	-
dermatofibrosarcoma protuberans	Gleevec	Orphan Designation	-
dermatomyositis	siponimod	Orphan Designation	-
diarrheal disease	Sandostatin LAR	Orphan Designation	-
diffuse large B-cell lymphoma	Kymriaheverolimus	Orphan Designation	FDA Approved
dilated cardiomyopathy 1X	Tafinlar(r) Capsules a nd Mekinist(r) Tablets	Orphan Designation	-
disease related to hematopoietic stem cell transplant	Spanlecortemlocel	Orphan Designation	-
disease related to solid organ transplantation	Simulect	Orphan Designation	-
ectopic ACTH secretion syndrome	Kymriah (tisagenlecleucel)	Orphan Designation	-
familial Mediterranean fever	ILARIS	Orphan Designation	-
familial Mediterranean fever, autosomal dominant	ILARIS	Orphan Designation	-
familial caudal dysgenesis	ILARIS	Orphan Designation	-
familial cold autoinflammatory syndrome 1	Ilaris	Orphan Designation	-
familial generalized lentiginosis	Sandostatin LAR	Orphan Designation	-
familial ovarian cancer	ILARIS	Orphan Designation	-
familial partial lipodystrophy, Kobberling type	selective deacylglycerol acyltransferase 1 inhibitor	Orphan Designation	-
fascioliasis	Egaten	Orphan Designation	-
fasciolopsiasis	Egaten	Orphan Designation	-
fetal cytomegalovirus syndrome	human IgG1 anti-human cytomegalovirus monoclonal antibodies LJP538 and LJP539	Orphan Designation	-
fibrochondrogenesis 1	Coartem	Orphan Designation	-
fibrous dysplasia	Sandostatin LAR	Orphan Designation	-
follicular lymphoma	Kymriah	Orphan Designation	FDA Approved
fungal lung infectious disease	Amphotericin B inhalation powder	Orphan Designation	-
gastric cancer	everolimus	Orphan Designation	-
gastrointestinal stromal tumor	Gleevec	Orphan Designation	-
glioma susceptibility 1	lapatinib ditosylate hydrochloridelapatinib	Orphan Designation	-
glioma susceptibility 2	MekinistTafinlar	Orphan Designation	-
glycogen storage disease VI	selective deacylglycerol acyltransferase 1 inhibitor	Orphan Designation	-
graft versus host disease	2-amino-2-[2-[2-chloro-4-[[3-(phenylmethoxy) phenyl] thio]phenyl]ethyl]1,3-propanediol hydrochloride	Orphan Designation	-
hematopoietic and lymphoid cell neoplasm	Spanlecortemlocel	Orphan Designation	-
hematopoietic and lymphoid system neoplasm	Spanlecortemlocel	Orphan Designation	-
hemimegalencephaly	Sandostatin LAR	Orphan Designation	-
hepatocellular carcinoma	everolimus	Orphan Designation	-
hereditary breast ovarian cancer syndrome	patupilone	Orphan Designation	-
homozygous 11P15-p14 deletion syndrome	inclisiran	Orphan Designation	-
homozygous familial hypercholesterolemia	inclisiran	Orphan Designation	-
humoral hypercalcemia of malignancy	Zometa, Zabel	Orphan Designation	-
hyper-IgE recurrent infection syndrome 1, autosomal dominant	Amphotericin B inhalation powder	Orphan Designation	-
hypertensive urgency	Simulect	Orphan Designation	-
idiopathic hypereosinophilic syndrome	Gleevec	Orphan Designation	-
immune dysregulation-polyendocrinopathy-enteropathy-X-linked syndrome	Sandostatin LAR	Orphan Designation	-
inclusion body myositis	bimagrumab	Orphan Designation	-
indolent systemic mastocytosis	Gleevec	Orphan Designation	-
intellectual disability, autosomal recessive 61	Ursodiol	Orphan Designation	-
intellectual disability-feeding difficulties-developmental delay-microcephaly syndrome	ZYKADIA	Orphan Designation	-
intestinal pseudoobstruction, neuronal, chronic idiopathic, X-linked	PromactaGleevec	Orphan Designation	-
leishmaniasis	parasite-selective proteasome inhibitor with potent activity against kinetoplastid parasites	Orphan Designation	-
local anesthetic poisoning	Tafinlar(r) Capsules a nd Mekinist(r) Tablets	Orphan Designation	-
lung adenocarcinoma	capmatinib	Orphan Designation	-
lung cancer	capmatinib	Orphan Designation	-
lymphatic malformation	Alpelisib	Orphan Designation	-
lymphatic malformation 5	Alpelisib	Orphan Designation	-
lymphatic malformation 6	Alpelisib	Orphan Designation	-
lymphoblastic leukemia, acute, with lymphomatous features	Arranon	Orphan Designation	-
lymphoblastic lymphoma	Arranon	Orphan Designation	-
lymphoplasmacytic lymphoma	everolimus	Orphan Designation	-
macroglobulinemia, Waldenstrom, 1	everolimus	Orphan Designation	-
major depressive disorder	TAFINLAR	Orphan Designation	-
malaria	Ganaplacide and Lumefantrine fixed-dose combinationcipargamin2-amino-1-[2-(4-fluoro-phenyl)-3-(4-fluoro-phenyl)amino)-8,8-dimethyl-5,6-dihydro-imidazo[1,2-a]pyrazin-7(8H)-yl)-ethanone diphosphate	Orphan Designation	-
malignant glioma	MekinistTafinlar	Orphan Designation	-
malignant pancreatic neoplasm	recombinant human anti-TGFBeta IgG2 monoclonal antibody that belongs to the IgG2/Gamma isotype subclass and binds TGFBeta1 and TGFBeta2 with high affinity and, to a lesser extent, TGFBeta3canakinumabspartalizumab	Orphan Designation	-
mastocytosis	Rydapt	Orphan Designation	-
medial condensing osteitis of the clavicle	Sandostatin LAR	Orphan Designation	-
medulloblastoma	sonidegib	Orphan Designation	-
melanoma of soft tissue	Votrient	Orphan Designation	-
methylmalonic acidemia with homocystinuria, type cblJ	Promacta	Orphan Designation	-
methylmalonic aciduria and homocystinuria type cblF	Promacta	Orphan Designation	-
micrognathia-recurrent infections-behavioral abnormalities-mild intellectual disability syndrome	ZYKADIA	Orphan Designation	-
mismatch repair cancer syndrome 1	MekinistTafinlar	Orphan Designation	-
mitochondrial DNA depletion syndrome 6 (hepatocerebral type)	Guanethidine monosulfate	Orphan Designation	-
mullerian aplasia and hyperandrogenism	patupilone	Orphan Designation	-
multiple congenital anomalies-hypotonia-seizures syndrome 2	Promacta	Orphan Designation	-
multiple sclerosis	MAYZENTGILENYA	-	FDA Approved
multisystemic smooth muscle dysfunction syndrome	Tafinlar(r) Capsules a nd Mekinist(r) TabletsMekinistpasireotideGleevec	Orphan Designation	-
myasthenia gravis	iptacopan	Orphan Designation	-
myasthenia, limb-girdle, autoimmune	ILARIS	Orphan Designation	-
mycosis fungoides	Simulect	Orphan Designation	-
myelodysplastic/myeloproliferative neoplasm	Gleevec	Orphan Designation	-
myeloproliferative disorder, chronic, with eosinophilia	Gleevec	Orphan Designation	-
myeloproliferative neoplasm	Gleevec	Orphan Designation	-
myeloproliferative neoplasm, unclassifiable	Gleevec	Orphan Designation	-
myopathy, epilepsy, and progressive cerebral atrophy	Sandostatin LAR	Orphan Designation	-
natal teeth-intestinal pseudoobstruction-patent ductus syndrome	Tafinlar(r) Capsules a nd Mekinist(r) TabletsMekinistdabrafenibpasireotideGleevec	Orphan Designation	-
necrobiosis lipoidica	ILARIS	Orphan Designation	-
neuroacanthocytosis	Kymriah (tisagenlecleucel)	Orphan Designation	-
neuroblastoma	ribociclib	Orphan Designation	-
neurodegeneration with brain iron accumulation 2A	Exjade; Jadenu Sprinkles	Orphan Designation	-
neurodevelopmental disorder with dysmorphic facies and thin corpus callosum	Gleevec	Orphan Designation	-
neurodevelopmental disorder with hypotonia and speech delay, with or without seizures	ZYKADIA	Orphan Designation	-
neurodevelopmental disorder with spasticity and poor growth	Gleevec	Orphan Designation	-
neuropathy, hereditary sensory and autonomic, adult-onset, with anosmia	Vijoice	Orphan Designation	-
nodular lichen myxedematosus	Afinitor	Orphan Designation	-
non-immunoglobulin-mediated membranoproliferative glomerulonephritis	Fabhalta	Orphan Designation	-
non-small cell lung carcinoma	capmatinib	Orphan Designation	-
non-small cell squamous lung carcinoma	capmatinib	Orphan Designation	-
nut midline carcinoma	Fabhalta	Orphan Designation	-
osteopathia striata with cranial sclerosis	PromactaILARIS	Orphan Designation	-
ovarian cancer	pazopanib	Orphan Designation	-
pancreatic neuroendocrine neoplasm	humanized monoclonal antibody against human PD-1octreotideAfinitor	Orphan Designation	-
pancreatic neuroendocrine tumor	humanized monoclonal antibody against human PD-1octreotideAfinitor	Orphan Designation	-
paroxysmal nocturnal hemoglobinuria	Fabhalta	Orphan Designation	-
pediatric acute-onset neuropsychiatric syndrome	MekinistTafinlarILARISTasigna	Orphan Designation	-
pediatric hepatocellular carcinoma	MekinistTafinlarILARISIlarisTasigna	Orphan Designation	-
pediatric high-grade glioma	MekinistTafinlar	Orphan Designation	-
pediatric ovarian dysgerminoma	MekinistTafinlarILARISTasigna	Orphan Designation	-
pediatric ovarian germ cell tumor	Tasigna	Orphan Designation	-
pediatric-onset Graves disease	MekinistTafinlarILARISIlarisTasigna	Orphan Designation	-
periodic fever syndrome	ILARIS	Orphan Designation	-
persistent hyperplastic primary vitreous, autosomal recessive	Tafinlar(r) Capsules a nd Mekinist(r) Tablets	Orphan Designation	-
plasma cell myeloma	a B-cell maturation antigen (BCMA)-directed, genetically engineered T cell immunotherapy product derived from an autologous leukapheresisDovitinib	Orphan Designation	-
polycystic kidney disease 1	Afinitor	Orphan Designation	-
polycystic kidney disease 2	a chemically modified full phosphorothioate-backbone anti-miR-17 oligonucleotideAfinitor	Orphan Designation	-
polymyositis	Siponimod	Orphan Designation	-
post 5-alpha-reductase inhibitors treatment syndrome	TAFINLAR	Orphan Designation	-
post-treatment Lyme disease syndrome	TAFINLAR	Orphan Designation	-
precursor T-cell acute lymphoblastic leukemia	Arranon	Orphan Designation	-
primary adult heart tumor	KymriahScemblixRydaptSigniforGleevec	Orphan Designation	-
primary cutaneous T-cell non-Hodgkin lymphoma	Panobinostat	Orphan Designation	-
primary peritoneal carcinoma	patupilone	Orphan Designation	-
primary peritoneal serous adenocarcinoma	patupilone	Orphan Designation	-
progressive familial intrahepatic cholestasis	Afinitor	Orphan Designation	-
progressive supranuclear palsy	Afinitor	Orphan Designation	-
progressive supranuclear palsy-pure akinesia with gait freezing syndrome	Afinitor	Orphan Designation	-
proteasome-associated autoinflammatory syndrome 6	Ilaris	Orphan Designation	-
proximal spinal muscular atrophy	Coartem	Orphan Designation	-
pulmonary arterial hypertension	imatinib	Orphan Designation	-
pulmonary non-tuberculous mycobacterial infection	clofazimine	Orphan Designation	-
renal cell carcinoma	VOTRIENT	-	FDA Approved
rhizomelic limb shortening with dysmorphic features	Gleevec	Orphan Designation	-
sickle cell disease	ADAKVEO	Orphan Designation	-
soft tissue sarcoma	Votrient	Orphan Designation	-
solid papillary breast carcinoma	Simulect	Orphan Designation	-
solid pseudopapillary carcinoma of pancreas	Simulect	Orphan Designation	-
spinal muscular atrophy, distal, autosomal recessive, 6	anti human Nogo-A human monoclonal antibody	Orphan Designation	-
spinal muscular atrophy, type II	branaplam	Orphan Designation	-
spinal muscular atrophy, type III	branaplam	Orphan Designation	-
spinal muscular atrophy, type IV	branaplam	Orphan Designation	-
squamous carcinoma in situ	MEKINIST and TAFINLARMEKINIST	Orphan Designation	-
stage II endometrioid carcinoma	spartalizumabMEKINIST and TAFINLARMEKINIST	Orphan Designation	-
stage IVb bladder cancer	spartalizumabMEKINIST and TAFINLARMEKINIST	Orphan Designation	-
sweet syndrome	Ilaris	Orphan Designation	-
systemic mastocytosis	Gleevec	Orphan Designation	-
tenosynovial giant cell tumor, diffuse type	Lacnotuzumab	Orphan Designation	-
treatment-refractory schizophrenia	TAFINLAR	Orphan Designation	-
tuberous sclerosis	Afinitor	Orphan Designation	-
tuberous sclerosis 1	Afinitor	Orphan Designation	-
tuberous sclerosis 2	Afinitor	Orphan Designation	-
tumor grade X, general grading system	Zometa, Zabel	Orphan Designation	-
tumor of adipose tissue	Zometa, Zabel	Orphan Designation	-
tumor of cranial and spinal nerves	Zometa, Zabel	Orphan Designation	-
tumor of salivary gland	Zometa, Zabel	Orphan Designation	-
tumor suppressor gene on chromosome 11	capmatinib	Orphan Designation	-
undifferentiated pleomorphic sarcoma	Votrient	Orphan Designation	-
uterine cervix carcinoma in situ	MEKINIST and TAFINLARMEKINIST	Orphan Designation	-
visceral neuropathy, familial, 2, autosomal recessive	lapatinib ditosylate hydrochloridelapatinib	Orphan Designation	-
vulvovaginal gingival syndrome	Ilaris	Orphan Designation	-

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