On 11 November 2015, orphan designation (EU/3/15/1580) was granted by the European Commission to Pharma Gateway AB, Sweden, for N-[5-(3,5-difluorobenzyl)-1H-indazol-3-yl]-4-(4 methylpiperazin-1-yl)-2-(tetrahydro-2H-pyran-4-ylamino)benzamide (also known as entrectinib) for the treatment of neuroblastoma.

This medicine is now known as entrectinib.

Please note that this product was withdrawn from the Community Register of designated Orphan Medicinal Products in December 2018 on request of the Sponsor.

Key facts

Active substance
N-[5-(3,5-difluorobenzyl)-1H-indazol-3-yl]-4-(4 methylpiperazin-1-yl)-2-(tetrahydro-2H-pyran-4-ylamino) benzamide (entrectinib)
Disease / condition
Treatment of neuroblastoma
Date of first decision
EU designation 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

Pharma Gateway AB
Johanneslundsvägen 2
194 61 Upplands Väsby
Tel. +46 8 59 07 78 00

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;

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