EU/3/19/2175

Table of contents

About

On 28 June 2019 orphan designation EU/3/19/2175 was granted by the European Commission to Spark Therapeutics Ireland Limited, Ireland, for recombinant adeno-associated viral vector containing a bioengineered capsid derived from AAV-rh74 and a codon-optimised expression cassette to drive the expression of a secretable form of human acid alpha-glucosidase (also known as SPK-3006) for the treatment of glycogen storage disease type II (Pompe's disease).

Key facts

Active substance
Recombinant adeno-associated viral vector containing a bioengineered capsid serotype AAV-rh74 and a codon-optimised expression cassette to drive the expression of a secretable form of human acid alpha-glucosidase
Disease / condition
Treatment of glycogen storage disease type II (Pompe's disease)
Date of first decision
28/06/2019
Outcome
Positive
EU designation number
EU/3/19/2175

Sponsor's contact details

Spark Therapeutics Ireland Limited
6th Floor
2 Grand Canal Quay
Dublin 2
Co. Dublin
Ireland
Tel. +353 87 2865356
E-mail: info@sparktx.com

Patients' organisations

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

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