EU/3/18/2071: Orphan designation for the treatment of medullary thyroid carcinoma



On 26 October 2018, orphan designation (EU/3/18/2071) was granted by the European Commission to Loxo Oncology Limited, United Kingdom, for 6-(2-hydroxy-2-methylpropoxy)-4-(6-(6-((6-methoxypyridin-3-yl)methyl)-3,6-diazabicyclo[3.1.1]heptan-3-yl)pyridin-3-yl)pyrazolo[1,5-a]pyridine-3-carbonitrile (also known as LOXO-292) for the treatment of medullary thyroid carcinoma.

The sponsorship was transferred to Eli Lilly Nederland B.V., Netherlands, in July 2019.

Please note that this product was withdrawn from the Union Register of orphan medicinal products in December 2019 on request of the Sponsor.

Key facts

Active substance
Intended use
Treatment of medullary thyroid carcinoma
Orphan designation status
EU designation number
Date of designation

Eli Lilly Nederland B.V.
Papendorpseweg 83
Utrecht 3528 BJ
The Netherlands
Tel. +31 3060 25800

Review of designation

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

Documents related to this orphan designation evaluation

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.

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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