R)-1-(3-(aminomethyl) phenyl)-N-(5-((3-cyanophenyl)(cyclopropylmethylamino)methyl)-2-fluorophenyl)-3-(trifluoromethyl)-1H-pyrazole-5-carboxamide dihydrochloride (berotralstat)


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.

Please note that this product (marketed as Orladeyo) was withdrawn from the Union Register of orphan medicinal products in March 2021 upon request of the marketing authorisation holder at the time of the granting of a marketing authorisation.

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)
Intented use
Treatment of hereditary angioedema
Date of designation
Orphan designation status
EU designation number

Review of designation

The Committee for Orphan Medicinal Products reviewed the orphan designation of the product 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 withdrawal assessment report – orphan maintenance.

Sponsor's contact details

Rocktwist House
Block 4
Harcourt Centre
Harcourt Road
Dublin 2
D02 HW77
Tel. +353 1223 3541

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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