EU/3/18/2014: Orphan designation for the treatment of Stargardt's disease

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 (Tinlarebant)


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 (Tinlarebant)
Intended use
Treatment of Stargardt's disease
Orphan designation status
EU designation number
Date of designation

MDC RegAffairs GmbH
Baumberger Strasse 11
50737 Cologne
North Rhine-Westphalia

Review of designation

The Committee for Orphan Medicinal Products reviews the orphan designation of a product if it is approved for marketing authorisation.

Update history

November 2022The sponsorship was transferred from IQVIA RDS Ireland Limited, Ireland, to MDC RegAffairs GmbH, Germany. 

Documents related to this orphan designation evaluation

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.

EU register of orphan medicines

The list of medicines that have received an orphan designation in the EU is available on the European Commission's website:

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