EU/3/19/2238

Table of contents

About

On 9 January 2020, orphan designation EU/3/19/2238 was granted by the European Commission to Esteve Pharmaceuticals S.A., Spain, for adeno-associated virus serotype 9 vector containing human n-acetylgalactosamine-6-sulfate sulfatase gene for the treatment of mucopolysaccharidosis type IVA (Morquio A Syndrome).

Key facts

Active substance
adeno-associated virus serotype 9 vector containing human n-acetylgalactosamine-6-sulfate sulfatase gene
Disease / condition
Treatment of mucopolysaccharidosis type IVA (Morquio A Syndrome)
Date of first decision
09/01/2020
Outcome
Positive
EU designation number
EU/3/19/2238

Sponsor's contact details

Esteve Pharmaceuticals S.A.
Torre Esteve
Passeig Zona Franca 109, 4º
08038 Barcelona
Spain
Tel. +34 93 4460 000
E-mail: evalenti@esteve.com

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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