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Table 1 Orphan-designated treatments meeting criteria for review

From: Review and comparison of clinical evidence submitted to support European Medicines Agency market authorization of orphan-designated oncological treatments

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)

  1. EU European Union