EU/3/07/514

About

On 4 December 2007, orphan designation (EU/3/07/514) was granted by the European Commission to Genzyme Europe BV, Netherlands, for (1R,2R)-octanoic acid[2-(2',3'-dihydro-benzo[1,4] dioxin-6'-yl)-2-hydroxy-1-pyrrolidin-1-ylmethyl-ethyl]-amide-L-tartaric acid salt for the treatment of Gaucher disease.

(1R,2R)-octanoic acid[2-(2',3'-dihydro-benzo[1,4] dioxin-6'-yl)-2-hydroxy-1-pyrrolidin-1-ylmethyl-ethyl]-amide-L-tartaric acid salt has been authorised in the EU as Cerdelga since 19 January 2015.

This medicine is now known as eliglustat.

Key facts

Active substance
(1R,2R)-octanoic acid[2-(2',3'-dihydro-benzo[1,4] dioxin-6'-yl)-2-hydroxy-1-pyrrolidin-1-ylmethyl-ethyl]-amide-L-tartaric acid salt (eliglustat)
Medicine name
Cerdelga
Disease / condition
Treatment of Gaucher disease
Date of decision
04/12/2007
Outcome
Positive
Orphan decision number
EU/3/07/514

Review of designation

During its meeting of 9 to 11 December 2014, the Committee for Orphan Medicinal Products (COMP) reviewed the designation EU/3/07/514 for Cerdelga (eliglustat)1 as an orphan medicinal product for the treatment of Gaucher disease. The COMP assessed whether, at the time of marketing authorisation, the medicinal product still met the criteria for orphan designation. The Committee looked at the seriousness and prevalence of the condition, and the existence of other methods of treatment. As other methods of treatment are authorised in the European Union (EU), the COMP also considered whether the medicine is of significant benefit to patients with Gaucher disease. The COMP recommended that the orphan designation of the medicine be maintained2.


1Previously known as (1R,2R)-octanoic acid[2-(2',3'-dihydro-benzo[1,4] dioxin-6'-yl)-2-hydroxy-1-pyrrolidin-1-ylmethyl-ethyl]-amide-L-tartaric acid salt.

2The maintenance of the orphan designation at time of marketing authorisation would, except in specific situations, give an orphan medicinal product 10 years of market exclusivity in the EU. This means that in the 10 years after its authorisation similar products with a comparable therapeutic indication cannot be placed on the market.

Sponsor's contact details

Genzyme Europe BV
Gooimeer 10
1411 DD Naarden
The Netherlands
Tel. +31 35 6991499
Fax +31 35 6991403
E-mail: eumedinfo@genzyme.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