EU/3/08/569: Orphan designation for the treatment of acute lymphoblastic leukaemia

Pegylated L-asparaginase

Table of contents


On 22 September 2008, orphan designation (EU/3/08/569) was granted by the European Commission to Enzon (UK) Limited, United Kingdom, for pegylated L-asparaginase for the treatment of acute lymphoblastic leukaemia.

The sponsorship was transferred to Defiante Farmacêutica, S.A., Portugal, in September 2010 and subsequently to Sigma-tau Rare Diseases S.A., Portugal, in November 2012.

Key facts

Active substance
Pegylated L-asparaginase
Intended use
Treatment of acute lymphoblastic leukaemia
Orphan designation status
EU designation number
Date of designation
Sigma-tau Rare Diseases S.A.
Rua dos Ferreiros, 260
9000-082 Funchal
Tel. +351 291 20 30 70
Fax +351 291 20 30 79

Documents related to this orphan designation evaluation

Patients' organisations

For contact details of patients’ organisations whose activities are targeted at rare diseases, see:

  • Orphanet, a database containing information on rare diseases, which includes a directory of patients’ organisations registered in Europe.

  • European Organisation for Rare Diseases (EURORDIS), a non-governmental alliance of patient organisations and individuals active in the field of rare diseases.

EU register of orphan medicines

The list of medicines that have received an orphan designation in the EU is available on the European Commission's website:

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