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Orphan designation

On 30 July 2002, orphan designation (EU/3/02/110) was granted by the European Commission to SciClone Pharmaceuticals Italy S.r.l., Italy, for thymalfasin for the treatment of hepatocellular carcinoma.

What is hepatocellular carcinoma?

Tumours that begin in the liver are known as liver tumours. Liver tumours which have the potential to grow rapidly and infiltrate healthy tissues are called hepatocellular carcinomas. Hepatocellular carcinoma is a life-threatening condition.

What are the methods of treatment available?

No medicinal products for the treatment of the condition had been authorised in the European Union at the time of submission of the application for orphan drug designation.

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

According to the information provided by the sponsor, hepatocellular carcinoma was considered to affect about 37,500 persons in the European Union.

How is this medicinal product expected to act?

Thymalfasin is a small protein. It was originally isolated from the thymus gland of cattle. Currently, this protein is produced by a process of chemical reactions. Thymalfasin acts on the immune system. This may increase the ability of immune cells called T-lymphocytes to kill tumour cells.

What is the stage of development of this medicinal product?

At the time of submission of the application for orphan designation, clinical trials in patients with hepatocellular carcinoma were ongoing.

In the European Union thymalfasin is authorised in Italy, where it is used to enhance the immune response stimulated by the influenza virus vaccine.

The sponsor has been granted orphan drug status for thymalfasin in treatment of hepatocellular carcinoma in the United States. 






Key facts

Product details for <p>Thymalfasin</p>
Active substanceThymalfasin
Medicine Name
Disease/conditionTreatment of hepatocellular carcinoma
Date of decision30/07/2002
Orphan decision numberEU/3/02/110

Review of designation