Generic name | Proprietary name | Orphan condition | Prevalence of orphan condition |
---|---|---|---|
Histamine dichloride | Ceplene | Adult acute myeloid leukaemia in first remission | 0.7/10,000 |
Decitabine | Dacogen | Adult acute myeloid leukaemia | <1/10,000 |
Pirfenidone | Esbriet | Adult mild to moderate idiopathic pulmonary fibrosis | ≤3/10,000 |
5-aminolevulinic acid | Gliolan | Visualization of malignant tissue during surgery for malignant glioma in adults | 1/10,000 |
Pomalidomide | Imnovid | Multiple myeloma | 2.2/10,000 |
Ruxolitinib | Jakavi | Splenomegaly or symptoms in adult patients with primary myelofibrosis | 0.5/10,000 |
Mifamurtide | Mepact | Osteosarcoma | 0.5/10,000 |
Sorafenib | Nexavar | Hepatocellular carcinoma; renal cell carcinoma | Hepatocellular carcinoma: 1/10,000; renal cell carcinoma: not reported |
Lenalidomide | Revlimid | Multiple myeloma | 1.3/10,000 |
Nilotinib | Tasigna | Adult patients with newly diagnosed Philadelphia-chromosome-positive chronic myelogenous leukaemia | 46,000 persons in EU (calculated as 1.0/10,000 based on EU population 457.7 million) |
Adult patients with Philadelphia-chromosome-positive chronic myelogenous leukaemia with resistance or intolerance to prior therapy including imatinib | |||
Thalidomide | Thalidomide Celgene | Untreated multiple myeloma in adults aged > 65 years or ineligible for high-dose chemotherapy | 1.2/10,000 |
Temsirolimus | Torisel | Renal cell carcinoma; adult patients with relapsed and/or refractory mantle cell lymphoma | Renal cell carcinoma: 3.5/10,000; refractory mantle cell lymphoma: 0.4/10,000 |
Azacitidine | Vidaza | Adults not eligible for haematopoietic transplant with myelodysplastic syndromes, chronic myelogenous leukaemia, and acute myeloid leukaemia | 1.1–3/10,000 |
Trabectedin | Yondelis | Treatment of patients with advanced soft tissue sarcoma | 23,000 persons in EU (calculated as 0.6/10,000 based on EU population 377 million) |