EU/3/15/1606: Orphan designation for the treatment of soft tissue sarcoma

(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)


Please note that this product (marketed as Vitrakvi) was withdrawn from the Community Register of designated orphan medicinal products in July 2019 upon request of the marketing authorisation holder at the time of the granting of a marketing authorisation.


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)
Intended use
Treatment of soft tissue sarcoma
Orphan designation status
EU designation number
Date of designation

Bayer AG
Kaiser-Wilhelm-Allee 1
51373 Leverkusen
Tel. +49 30 300139003

Review of designation

The Committee for Orphan Medicinal Products reviewed the orphan designation of Vitrakvi at the time of marketing authorisation.

The sponsor formally requested the withdrawal of the orphan designation prior to the Committee’s final opinion. More information is available in the PDF icon orphan designation withdrawal assessment report .

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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