Table 2.
Rare disorders with an approved OMP in EU until 31 December 2008
| Rare disorder (N = 44) |
First OMP (INN-name) |
Other ODs* (N = 149) |
|---|---|---|
| Fabry disease | agalsidase alfa/agalsidase beta** | 1 |
| Chronic myeloid leukemia | imatinib | 7 |
| Gaucher disease | miglustat | 3 |
| Acute promyelocytic leukemia | arsenic trioxide | 2 |
| Pulmonary arterial hypertension | bosentan | 12 |
| Acromegaly | pegvisomant | 2 |
| Gastrointestinal stromal tumors | imatinib | 3 |
| Familial adenomatous polypopsis | celecoxib | 2 |
| NAGS-deficiency | carglumic acid | - |
| Treatment prior to hematopoietic progenitor cell | ||
| transplantation | busulfan | - |
| Mucopolysaccharidosis type I | laronidase | - |
| Essential thrombocythaemia | anagrelide | - |
| Wilson's disease | zinc acetate dihydrate | 1 |
| High-grade dysplasia in Barrett's oesophagus | porfimer sodium | - |
| Patent ductus arteriosus | ibuprofen | - |
| Adrenal cortical carcinoma | mitotane | 1 |
| Hairy cell leukemia | cladribine | 1 |
| Narcolepsy-cataplexy | sodium oxybate | 1 |
| Chronic pain requiring intraspinal analgesia | ziconotide | - |
| Tyrosinaemia type I (hereditary) | nitisinone | - |
| Dermatofibrosarcoma protuberans | imatinib | - |
| Acute lymphoblastic leukemia | clofarabine | 16 |
| Anthracycline extravasations | dexrazoxane | - |
| Renal cell carcinoma | sorafenib | 18 |
| Mucopolysaccharidosis type VI | galsulfase | - |
| Glycogen storage disease type II | alglucosidase alpha | 1 |
| Chronic iron overload requiring chelation therapy | deferasirox | 2 |
| Hypereosinophilic syndrome | imatinib | 1 |
| Myelodysplastic syndromes | imatinib | 7 |
| Hepatocellular carcinoma | sorafenib | 9 |
| Soft tissue sarcoma | trabectedin | 7 |
| Paroxysmal nocturnal haemoglobinuria | eculizumab | - |
| Sickle cell syndrome | hydroxycarbamide | 3 |
| Multiple myeloma | lenalidomide | 14 |
| Lennox-Gastaut syndrome | rufinamide | - |
| Severe primary IGF-1 deficiency | mecasermin | - |
| Mucopolysaccharidosis type II | idursulfase | - |
| Severe myoclonic epilepsy in infancy (Dravet's syndrome) | stiripentol | - |
| Homocystinuria | betaine anhydrous | - |
| Systemic sclerosis | bosentan | 3 |
| Chronic myelomonocytic leukemia | azacitidine | - |
| Hyperphenylalaninaemia | sapropterin dihydrochloride | 2 |
| Angioedema (hereditary) | icatibant | 3 |
| Acute myeloid leukemia | histamine dihydrochloride | 27 |
* Other ODs in the EU under development for treatment of the specific rare disorder between the start of the EU Orphan Drug Regulation (April 2000) and 30 April 2010.
** Both agalsidase alfa and agalsidase beta obtained market approval at the 3th of August 2001 in the EU.