EU/3/16/1806

About

On 12 December 2016, orphan designation (EU/3/16/1806) was granted by the European Commission to Ser-mes Planificación SL, Spain, for recombinant self-complementary adeno-associated viral vector serotype 9 containing the human CLN3 gene (also known as ABO-201) for the treatment of neuronal ceroid lipofuscinosis.

Key facts

Active substance
Recombinant self-complementary adeno-associated viral vector serotype 9 containing the human CLN3 gene
Disease / condition
Treatment of neuronal ceroid lipofuscinosis
Date of decision
08/10/2009
Outcome
Positive
Orphan decision number
EU/3/16/1806

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

Abeona Therapeutics Europe SL
Calle Manoteras 30, A207-208
28050 Madrid
Spain
Tel. + 34 685 895 069
E-mail: abeonaeu@abeonatherapeutics.com

Patients' organisations

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

  • Orphanet, a database containing information on rare diseases, which includes a directory of patients’ organisations registered in Europe;
  • European Organisation for Rare Diseases (EURORDIS), a non-governmental alliance of patient organisations and individuals active in the field of rare diseases.

How useful was this page?

Add your rating