A biopharmaceutical company engaged in developing treatments for rare diseases, with 22 orphan drug designations and active involvement in 67 rare conditions, including pediatric lupus and idiopathic pulmonary fibrosis.
22
Orphan Designations
38
FDA Approvals
67
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| 3M syndrome 1 | Humatrope | Orphan Designation | - |
| Adams-Oliver syndrome 1 | Evista | Orphan Designation | - |
| Becker muscular dystrophy | tadalafil | Orphan Designation | - |
| Bicervical bicornuate uterus with patent cervix and vagina | CYRAMZAAlimta | Orphan Designation | - |
| Char syndrome | CYRAMZAAlimta | Orphan Designation | - |
| Diamond-Blackfan anemia 11 | Evista | Orphan Designation | - |
| Duchenne muscular dystrophy | tadalafil | Orphan Designation | - |
| Gorham-Stout disease | Evista | Orphan Designation | - |
| IgE responsiveness, atopic | Evista | Orphan Designation | - |
| Kocher-debre-Semelaigne syndrome | Humatrope | Orphan Designation | - |
| Noonan syndrome 3 | olomorasib | Orphan Designation | - |
| adult neuronal ceroid lipofuscinosis | Lartruvo | Orphan Designation | - |
| adult pleomorphic rhabdomyosarcoma | Lartruvo | Orphan Designation | - |
| adult-onset non-insulinoma persistent hyperinsulinemic hypoglycemia | Lartruvo | Orphan Designation | - |
| adult-onset proximal spinal muscular atrophy, autosomal dominant | Lartruvo | Orphan Designation | - |
| arteriovenous malformations of the brain | olomorasib | Orphan Designation | - |
| biliary tract cancer | merestinib | Orphan Designation | - |
| cancer-associated retinopathy | tasisulam sodium | Orphan Designation | - |
| carbon monoxide-induced delayed encephalopathy | necitumumabCYRAMZAAlimta | Orphan Designation | - |
| cardiofaciocutaneous syndrome 2 | olomorasib | Orphan Designation | - |
| cervical non-keratinizing squamous cell carcinoma | necitumumab | Orphan Designation | - |
| cutaneous melanoma | tasisulam sodium | Orphan Designation | - |
| diabetic ketoacidosis | REZVOGLAR KWIKPENBASAGLAR TEMPO PEN | - | FDA Approved |
| discrimination, Two-point, reduction 1N | Evista | Orphan Designation | - |
| drug- or toxin-induced pulmonary arterial hypertension | Adcirca | Orphan Designation | - |
| ectopia pupillae | Lartruvo | Orphan Designation | - |
| erectile dysfunction | CIALIS | - | FDA Approved |
| familial pulmonary arterial hypertension leucopenia and atrial septal defect | Adcirca | Orphan Designation | - |
| fibrous dysplasia | Evista | Orphan Designation | - |
| gastric cancer | CYRAMZA | Orphan Designation | - |
| glioma | transforming growth factor-beta receptor 1 kinase inhibitor | Orphan Designation | - |
| growth hormone insensitivity syndrome | Humatrope | Orphan Designation | - |
| hemoglobin--variants for which the chain carrying the mutation 1S unknown or uncertain | Lartruvo | Orphan Designation | - |
| hepatocellular carcinoma | transforming growth factor-beta receptor 1 kinase inhibitorCYRAMZA | Orphan Designation | - |
| heritable pulmonary arterial hypertension | Adcirca | Orphan Designation | - |
| idiopathic pulmonary arterial hypertension | Adcirca | Orphan Designation | - |
| idiopathic pulmonary fibrosis | lebrikizumab | Orphan Designation | - |
| laryngo-onycho-cutaneous syndrome | Humatrope | Orphan Designation | - |
| malignant pancreatic neoplasm | pegylated recombinant human interleukin-10 | Orphan Designation | - |
| malignant pleural mesothelioma | Alimta | Orphan Designation | - |
| malocclusion and short stature | Humatrope | Orphan Designation | - |
| medial condensing osteitis of the clavicle | Evista | Orphan Designation | - |
| melanoma | tasisulam sodium | Orphan Designation | - |
| microcephaly, short stature, and impaired glucose metabolism 2 | Humatrope | Orphan Designation | - |
| multisystemic smooth muscle dysfunction syndrome | CYRAMZAAlimta | Orphan Designation | - |
| myelodysplastic/myeloproliferative neoplasm | Adenosine triphosphate type 1 competitive inhibitor of JAK2 V617F tyrosine kinase | Orphan Designation | - |
| myeloproliferative disorder, chronic, with eosinophilia | Adenosine triphosphate type 1 competitive inhibitor of JAK2 V617F tyrosine kinase | Orphan Designation | - |
| myeloproliferative neoplasm | Adenosine triphosphate type 1 competitive inhibitor of JAK2 V617F tyrosine kinase | Orphan Designation | - |
| natal teeth-intestinal pseudoobstruction-patent ductus syndrome | CYRAMZAAlimta | Orphan Designation | - |
| neuroblastoma | (2R,4R)-1-(3-chloro-2-fluorobenzyl)-4-((3-fluoro-6-((5-methyl-1H-pyrazol-3-yl)amino)pyridin-2-yl)methyl)-2-methylpiperidine-4-carboxylic acid; 2- methylpropan-2-amine | Orphan Designation | - |
| non-keratinizing sinonasal squamous cell carcinoma | necitumumab | Orphan Designation | - |
| non-small cell squamous lung carcinoma | necitumumab | Orphan Designation | - |
| nut midline carcinoma | Humatrope | Orphan Designation | - |
| pediatric systemic lupus erythematosus | baricitinib | Orphan Designation | - |
| pituitary hormone deficiency, combined, 1 | Humatrope | Orphan Designation | - |
| plasma cell myeloma | tabalumab | Orphan Designation | - |
| primary adult heart tumor | Lartruvo | Orphan Designation | - |
| pulmonary arterial hypertension | Adcirca | Orphan Designation | - |
| ravine syndrome | Lartruvo | Orphan Designation | - |
| recessive dystrophic epidermolysis bullosa-generalized other | necitumumab | Orphan Designation | - |
| short stature, Brussels type | Humatrope | Orphan Designation | - |
| short stature-pituitary and cerebellar defects-small sella turcica syndrome | Humatrope | Orphan Designation | - |
| skeletal dysplasia, mild, with joint laxity and advanced bone age | CYRAMZA | Orphan Designation | - |
| skeletal dysplasia-epilepsy-short stature syndrome | Humatrope | Orphan Designation | - |
| soft tissue sarcoma | Lartruvo | Orphan Designation | - |
| trichohepatoenteric syndrome 1 | Humatrope | Orphan Designation | - |
| vulvar non-keratinizing squamous cell carcinoma | necitumumab | Orphan Designation | - |