EU/3/18/2028

About

This medicine is now known as berotralstat.

On 27 June 2018, orphan designation (EU/3/18/2028) was granted by the European Commission to BioCryst UK Ltd, United Kingdom, for (R)-1-(3-(aminomethyl) phenyl)-N-(5-((3-cyanophenyl)(cyclopropylmethylamino)methyl)-2-fluorophenyl)-3-(trifluoromethyl)-1H-pyrazole-5-carboxamide dihydrochloride (also known as BCX7353) for the treatment of hereditary angioedema.

The sponsorship was transferred to Biocryst Ireland Limited in March 2020.

Key facts

Active substance
R)-1-(3-(aminomethyl) phenyl)-N-(5-((3-cyanophenyl)(cyclopropylmethylamino)methyl)-2-fluorophenyl)-3-(trifluoromethyl)-1H-pyrazole-5-carboxamide dihydrochloride (berotralstat)
Disease / condition
Treatment of hereditary angioedema
Date of first decision
27/06/2018
Outcome
Positive
EU designation number
EU/3/18/2028

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

Rocktwist House
Block 1
Western Business Park
Shannon
Co. Clare
Ireland
Tel. +353 1223 3541
E-mail: safety@biocryst.com

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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