11
Orphan Designations
0
FDA Approvals
30
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| B-cell chronic lymphocytic leukemia | GAZYVA | Orphan Designation | - |
| Bicervical bicornuate uterus with patent cervix and vagina | GAZYVA | Orphan Designation | - |
| Char syndrome | GAZYVA | Orphan Designation | - |
| Ehlers-Danlos syndrome, kyphoscoliotic type, 2 | GAZYVA | Orphan Designation | - |
| Muckle-Wells syndrome | N-[[(1,2,3,5,6,7-hexahydro-s-indacen-4-yl) amino]carbonyl]-1-(1-methylethyl)-1H-Pyrazole-3-sulfonamide | Orphan Designation | - |
| acute myeloid leukemia | venetoclax + idasanutlin | Orphan Designation | - |
| anti-HLA hyperimmunization | satralizumab-mwge | Orphan Designation | - |
| anti-glomerular basement membrane disease | satralizumab-mwge | Orphan Designation | - |
| anti-neutrophil antibody associated vasculitis | satralizumab-mwge | Orphan Designation | - |
| autosomal recessive distal spinal muscular atrophy 1 | Evrysdi | Orphan Designation | - |
| carbon monoxide-induced delayed encephalopathy | GAZYVA | Orphan Designation | - |
| cryopyrin-associated periodic syndrome | N-[[(1,2,3,5,6,7-hexahydro-s-indacen-4-yl) amino]carbonyl]-1-(1-methylethyl)-1H-Pyrazole-3-sulfonamide | Orphan Designation | - |
| diffuse large B-cell lymphoma | obinutuzumab | Orphan Designation | - |
| familial cold autoinflammatory syndrome 1 | N-[[(1,2,3,5,6,7-hexahydro-s-indacen-4-yl) amino]carbonyl]-1-(1-methylethyl)-1H-Pyrazole-3-sulfonamide | Orphan Designation | - |
| follicular lymphoma | GAZYVA | Orphan Designation | - |
| glycogen storage disease II | recombinant adeno-associated viral (AAV) vector that contains a bio-engineered capsid (AAV-Spark100) and a codon-optimized expression cassette to drive expression of a secretable form of human acid a-glucosidase (GAA) | Orphan Designation | - |
| in situ carcinoma | GAZYVA | Orphan Designation | - |
| membranous glomerulonephritis | obinutuzumab | Orphan Designation | - |
| multisystemic smooth muscle dysfunction syndrome | GAZYVA | Orphan Designation | - |
| natal teeth-intestinal pseudoobstruction-patent ductus syndrome | GAZYVA | Orphan Designation | - |
| primary pulmonary diffuse large B-cell lymphoma | obinutuzumab | Orphan Designation | - |
| skin carcinoma in situ | GAZYVA | Orphan Designation | - |
| spinal muscular atrophy | Evrysdi | Orphan Designation | - |
| spinal muscular atrophy with congenital bone fractures 1 | Evrysdi | Orphan Designation | - |
| spinal muscular atrophy with congenital bone fractures 2 | Evrysdi | Orphan Designation | - |
| spinal muscular atrophy, type IV | Evrysdi | Orphan Designation | - |
| splenic marginal zone lymphoma | obinutuzumab | Orphan Designation | - |
| stomach carcinoma in situ | GAZYVA | Orphan Designation | - |
| tetragametic chimerism | Actemra | Orphan Designation | - |
| thyroid gland diffuse large B-cell lymphoma | obinutuzumab | Orphan Designation | - |