EU/3/09/661

About

On 30 May 2016, orphan designation (EU/3/09/661) was granted by the European Commission to Ipsen Pharma, France, for (S)-ethyl 2-amino-3-(4-(2-amino-6(-1-(4-chloro-2-(3-methyl-1H-pyrazol-1-yl)phenyl)-2,2,2-trifluoroethoxy)pyrimidin-4-yl)phenyl)propanoate (also known as telotristat etiprate) for the treatment of carcinoid syndrome.

(S)-ethyl 2-amino-3-(4-(2-amino-6(-1-(4-chloro-2-(3-methyl-1H-pyrazol-1-yl)phenyl)-2,2,2-trifluoroethoxy)pyrimidin-4-yl)phenyl)propanoate has been authorised in the EU as Xermelo since 18 September 2017.

Key facts

Active substance
(S)-ethyl 2-amino-3-(4-(2-amino-6((R)-1-(4-chloro-2-(3-methyl-1H-pyrazol-1-yl)phenyl)-2,2,2-trifluoroethoxy)pyrimidin-4-yl)phenyl)propanoate
Medicine name
Xermelo
Disease / condition
Treatment of carcinoid syndrome
Date of decision
30/05/2016
Outcome
Positive
Orphan decision number
EU/3/09/661

Review of designation

On 25 July 2017, the Committee for Orphan Medicinal Products (COMP) completed a review of the designation EU/3/09/661 for Xermelo (telotristat1) as an orphan medicinal product for the treatment of carcinoid syndrome. 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 carcinoid syndrome. The COMP recommended that the orphan designation of the medicine be maintained2.


1Previously known as (S)-ethyl 2-amino-3-(4-(2-amino-6(-1-(4-chloro-2-(3-methyl-1H-pyrazol-1-yl)phenyl)-2,2,2-trifluoroethoxy)pyrimidin-4-yl)phenyl)propanoate.

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 the same therapeutic indication cannot be placed on the market.

Sponsor's contact details

Ipsen Pharma
65 Quai Georges Gorse
92100 Boulogne-Billancourt
France
Tel. +33 1 58 33 50 00
E-mail: http://www.ipsen.com/en/contact-us/

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.

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