EU/3/18/2015

About

On 25 May 2018, orphan designation (EU/3/18/2015) was granted by the European Commission to Baxalta Innovations GmbH, Austria, for adeno-associated viral vector serotype 8 containing a functional copy of the codon-optimised F8 cDNA encoding the B-domain deleted human coagulation factor VIII for the treatment of haemophilia A.

Key facts

Active substance
Adeno-associated viral vector serotype 8 containing a functional copy of the codon-optimised F8 cDNA encoding the B-domain deleted human coagulation factor VIII
Disease / condition
Treatment of haemophilia A
Date of first decision
25/05/2018
Outcome
Positive
EU designation number
EU/3/18/2015

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

Baxalta Innovations GmbH
Industriestrasse 67
A-1221 Vienna
Austria
Tel. +43 1 20 100 0
Fax +43 1 20 100 247 5990
E-mail: EU.RA.Info@baxalta.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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