On 11 January 2016, orphan designation (EU/3/15/1606) was granted by the European Commission to TMC Pharma Services Ltd, United Kingdom, for (S)-N-(5-((R)-2-(2,5-difluorophenyl)pyrrolidin-1-yl)pyrazolo[1,5-a]pyrimidin-3-yl)-3-hydroxypyrrolidine-1-carboxamide hydrogen sulfate (also known as LOXO-101) for the treatment of soft tissue sarcoma.

The sponsorship was transferred to Loxo Oncology Limited, United Kingdom in November 2017.

This medicine is now known as larotrectinib.

The sponsorship was transferred to Bayer AG - Germany, in June 2018.

Key facts

Active substance
(S)-N-(5-((R)-2-(2,5-difluorophenyl)pyrrolidin-1-yl)pyrazolo[1,5-a]pyrimidin-3-yl)-3-hydroxypyrrolidine-1-carboxamide hydrogen sulfate (larotrectinib)
Disease / condition
Treatment of soft tissue sarcoma
Date of decision
Orphan decision number

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

Bayer AG
Kaiser-Wilhelm-Allee 3
51368 Leverkusen

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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