EU/3/18/2014

About

On 25 May 2018, orphan designation (EU/3/18/2014) was granted by the European Commission to IQVIA RDS Ireland Limited, Ireland, for 1-(3-{4-[3,4-difluoro-2-(trifluoromethyl)phenyl]piperidine-1-carbonyl}-1H,4H,5H,6H,7H-pyrazolo[3,4-c]pyridin-6-yl)ethan-1-one (also known as LBS-008) for the treatment of Stargardt's disease.

Key facts

Active substance
1-(3-{4-[3,4-difluoro-2-(trifluoromethyl)phenyl]piperidine-1-carbonyl}-1H,4H,5H,6H,7H-pyrazolo[3,4- c]pyridin-6-yl)ethan-1-one
Disease / condition
Treatment of Stargardt's disease
Date of first decision
25/05/2018
Outcome
Positive
EU designation number
EU/3/18/2014

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

IQVIA RDS Ireland Limited
Estuary House
East Point Business Park
Fairview
Dublin 3
Ireland
Tel. +353 1 819 51 00
Fax +353 1 819 95 11
E-mail: cliona.oleary@iqvia.com, mohit.kashyap@iqvia.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