Table 2 Rare disorders with an approved OMP in EU until 31 December 2008

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