EU/3/18/2048

About

On 31 July 2018, orphan designation (EU/3/18/2048) was granted by the European Commission to Pharma Gateway AB, Sweden, for N-acetylgalactosamine-conjugated synthetic double-stranded oligomer specific to serpin family A member 1 gene (also known as ARO-AAT) for the treatment of congenital alpha-1 antitrypsin deficiency.

Key facts

Active substance
N-acetylgalactosamine-conjugated synthetic double-stranded oligomer specific to serpin family A member 1 gene
Disease / condition
Treatment of congenital alpha-1 antitrypsin deficiency
Date of decision
31/07/2018
Outcome
Positive
Orphan decision number
EU/3/18/2048

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

Pharma Gateway AB
Johanneslundsvägen 2
Oxfordhuset
194 81 Upplands Väsby
Sweden
Tel. +46 8590 77800
Fax +46 8590 71440
E-mail: info@pharmagateway.eu

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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