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.

The sponsor’s address was updated in November 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 4
Harcourt Centre
Harcourt Road
Dublin 2
D02 HW77
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.

How useful was this page?

Add your rating