EU/3/19/2156

About

On 24 April 2019, orphan designation (EU/3/19/2156) was granted by the European Commission to Pharma Gateway AB, Sweden, for modified messenger ribonucleic acid encoding human propionyl-coenzyme A carboxylase alpha and beta subunits encapsulated into lipid nanoparticle (also known as mRNA-3927) for the treatment of propionic acidaemia.

Key facts

Active substance
Modified messenger ribonucleic acid encoding human propionyl-coenzyme A carboxylase alpha and beta subunits encapsulated into lipid nanoparticle
Disease / condition
Treatment of propionic acidaemia
Date of first decision
24/04/2019
Outcome
Positive
EU designation number
EU/3/19/2156

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
Johanneslundsvagen 2
Hammarby
194 61 Upplands Vaesby
Stockholms Lan
Sweden
Tel: +1 617 209 5956 (USA)
E-mail: christine.swenson@modernatx.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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