A biopharmaceutical company focused on developing treatments for rare diseases, with 19 FDA-approved drugs and 23 orphan drug designations across 42 rare disease indications.
23
Orphan Designations
19
FDA Approvals
42
Rare Diseases
9
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| ATTRV30M amyloidosis | coramitug | Orphan Designation | - |
| COG4-congenital disorder of glycosylation | Norditropin | Orphan Designation | - |
| Noonan syndrome 1 | RebinynCoagulation Factor VIIa (Recombinant)NorditropinTRETTEN | Orphan Designation | - |
| Noonan syndrome-like disorder with loose anagen hair 2 | Norditropin | Orphan Designation | - |
| RFT1-congenital disorder of glycosylation | NovoSevenCoagulation factor VIIa (recombinant)NovoSeven RT | Orphan Designation | - |
| Stormorken syndrome | NovoSevenCoagulation factor VIIa (recombinant)NovoSeven RT | Orphan Designation | - |
| Wolcott-Rallison syndrome | anti-interleukin 21 (NNC0114-0006) in combination with liraglutide | Orphan Designation | - |
| acroosteolysis dominant type | Norditropin | Orphan Designation | - |
| arthrogryposis, renal dysfunction, and cholestasis 1 | RebinynCoagulation Factor VIIa (Recombinant)TRETTEN | Orphan Designation | - |
| autism | AlhemoRebinyn | Orphan Designation | - |
| bleeding disorder, platelet-type, 21 | TRETTEN | Orphan Designation | - |
| bleeding disorder, platelet-type, 22 | NovoSevenNovoSeven RTTRETTEN | Orphan Designation | - |
| bleeding disorder, platelet-type, 25 | TRETTEN | Orphan Designation | - |
| cerebrooculofacioskeletal syndrome 4 | Norditropin | Orphan Designation | - |
| congenital | Factor XIII [A2] homodimer, recombinant DNA origin | Orphan Designation | - |
| congenital adrenal hyperplasia | Factor XIII [A2] homodimer, recombinant DNA origin | Orphan Designation | - |
| congenital heart disease | Factor XIII [A2] homodimer, recombinant DNA origin | Orphan Designation | - |
| esophageal varices | Rebinyn | Orphan Designation | - |
| familial amyloid neuropathy | coramitug | Orphan Designation | - |
| glycogen storage disease VI | Norditropin | Orphan Designation | - |
| hemophilia A | recombinant bispecific human IgG4 antibody which functionally mimics the procoagulant properties of activated FVIIIAlhemo | Orphan Designation | - |
| hemophilia B | Alhemo | Orphan Designation | - |
| hemorrhagic disease of newborn | RebinynCoagulation Factor VIIa (Recombinant)NovoSevenTRETTEN | Orphan Designation | - |
| immune dysregulation-polyendocrinopathy-enteropathy-X-linked syndrome | anti-interleukin 21 (NNC0114-0006) in combination with liraglutide | Orphan Designation | - |
| inflammatory bowel disease 1 | Norditropin | Orphan Designation | - |
| intellectual developmental disorder, X-linked, syndromic, Pilorge type | AlhemoRebinyn | Orphan Designation | - |
| intellectual disability-severe speech delay-mild dysmorphism syndrome | AlhemoRebinyn | Orphan Designation | - |
| juvenile-onset diabetes mellitus-central and peripheral neurodegeneration syndrome | anti-interleukin 21 (NNC0114-0006) in combination with liraglutide | Orphan Designation | - |
| laryngo-onycho-cutaneous syndrome | Norditropin | Orphan Designation | - |
| nut midline carcinoma | Norditropin | Orphan Designation | - |
| peripheral primitive neuroectodermal tumor | NovoSevenNovoSeven RT | Orphan Designation | - |
| platelet-type bleeding disorder 19 | Rebinyn | Orphan Designation | - |
| sickle cell disease | decitabine and tetrahydrouridine | Orphan Designation | - |
| sitosterolemia 1 | NovoSevenCoagulation factor VIIa (recombinant)NovoSeven RT | Orphan Designation | - |
| spinal muscular atrophy | Rebinyn | Orphan Designation | - |
| thalassemia | etavopivat5-aza-2'-dexocytidine combined with 1-[(2R,3R,4S,5R)-3,4-dihydroxy-5-(hydroxymethyl)oxolan-2-yl]-4-hydroxy-1,3-diazinan-2-one | Orphan Designation | - |
| type 1 diabetes mellitus 1 | OZEMPIC | - | FDA Approved |
| type 1 diabetes mellitus 11 | OZEMPIC | - | FDA Approved |
| type 1 diabetes mellitus 12 | OZEMPIC | - | FDA Approved |
| type 1 diabetes mellitus 13 | OZEMPIC | - | FDA Approved |
| type 1 diabetes mellitus 15 | OZEMPIC | - | FDA Approved |
| type 2 diabetes mellitus | RYBELSUS | - | FDA Approved |