EU/3/18/2049

About

On 31 July 2018, orphan designation (EU/3/18/2049) was granted by the European Commission to Inozyme Pharma Ireland Ltd, Ireland, for recombinant human ectonucleotide pyrophosphatase/phosphodiesterase 1 fused to the Fc fragment of IgG1 (also known as INZ-701) for the treatment of ectonucleotide pyrophosphatase/phosphodiesterase 1 deficiency.

Key facts

Active substance
recombinant human ectonucleotide pyrophosphatase/phosphodiesterase 1 fused to the Fc fragment of IgG1
Disease / condition
treatment of ectonucleotide pyrophosphatase/phosphodiesterase 1 deficiency
Date of decision
31/07/2018
Outcome
Positive
Orphan decision number
EU/3/18/2049

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

Inozyme Pharma Ireland Ltd
29 Earlsfort Terrace
Dublin 2
Ireland
Tel. +353 1 485 4030
E-mail: info@inozyme.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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