Summary of opinion on orphan designation

summary of opinion on orphan designation

3-(4'aminoisoindoline-1'-one)-1-piperidine-2,6-dione for the treatment of myelodysplastic syndromes

On 8 March 2004, orphan designation (EU/3/04/192) was granted by the European Commission to Gregory Fryer Associates Limited, United Kingdom, for 3-(4'aminoisoindoline-1'-one)-1-piperidine-2,6-dione for the treatment of myelodysplastic syndromes.

The sponsorship was transferred to Celgene Europe Limited in July 2005.

What are myelodysplastic syndromes?

Myelodysplastic syndromes are a distinct group of disorders in which the production of blood cells by the bone marrow is abnormal. The bone marrow is the spongy tissue found in the large bones. It has the function of producing red cells (which are the main carriers of oxygen to body tissues), white blood cells (which fight infection), and platelets (which make the blood clot). In myelodysplastic syndromes these cells do not grow and maturate normally. Consequently several symptoms can develop: fatigue or weakness (due to anaemia, the red cells deficit), infections (due to decrease in white blood cells) or easy bruising or abnormal bleeding (platelets deficit). Myelodysplastic syndromes are life threatening because it can result in severe anaemia, infections or haemorrhages and it can progress to acute leukaemia.

What is the estimated number of patients affected by the condition?

At the time of designation, myelodysplastic syndromes affected between 1 and 3 in 10,000 people in the European Union (EU)*. This is equivalent to a total of around 39,000 to 116,000 people, and is below the ceiling for orphan designation, which is 5 people in 10,000. This is based on the information provided by the sponsor and the knowledge of the Committee for Orphan Medicinal Products (COMP).

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