On 21 June 2011, orphan designation (EU/3/11/877) was granted by the European Commission to Laboratorios del Dr. Esteve, S.A., Spain, for adeno-associated viral vector serotype 9 containing the human sulfamidase gene for the treatment of mucopolysaccharidosis type IIIA (Sanfilippo A syndrome).

In September 2018, Laboratorios del Dr.Esteve, S.A changed name to Esteve Pharmaceuticals, S.A. The sponsor's address was updated in September 2018.

Key facts

Active substance
Adeno-associated viral vector serotype 9 containing the human sulfamidase gene
Disease / condition
Treatment of mucopolysaccharidosis, type IIIA (Sanfilippo A syndrome)
Date of first decision
EU designation number

Review of designation

The Committee for Orphan Medicinal Products reviews the orphan designation of a product if it is approved for marketing authorisation.

Sponsor's contact details

Esteve Pharmaceuticals, S.A.
Passeig de la Zona Franca, 109
4th floor
08038 Barcelona
Tel.: +34 93 446 60 00

Patients' organisations

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

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

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

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