MedPath
EMA Approval

Orladeyo

B06AC

用于遗传性血管性水肿的药物

Other hematological agents

berotralstat

Angioedemas, Hereditary

berotralstat dihydrochloride

Basic Information

EMA regulatory identification and product classification information

EMA Identifiers

ATC CodeB06AC
EMA European Classification

Overview Summary

Comprehensive product overview and regulatory summary

Orladeyo is a medicine used to prevent attacks of hereditary angioedema (swelling) in patients from 12 years of age. Patients with hereditary angioedema have attacks of rapid swelling such as in the face, throat, arms and legs, or around the gut.

Hereditary angioedema is rare, and Orladeyo was designated an ‘orphan medicine’ (a medicine used in rare diseases) on 27 June 2018. Further information on the orphan designation can be found here.

Orladeyo contains the active substance berotralstat.

Authorisations (1)

EMEA/H/C/005138

BioCryst Ireland Limited,Rocktwist House, Block 1,Western Business Park,Shannon Co.,Clare V14 FW97,Ireland

Authorised

April 30, 2021

Active Substances (1)

berotralstat dihydrochloride

Documents (14)

Orladeyo: EPAR - Product information

June 1, 2021

DRUG_PRODUCT_INFORMATION

Orladeyo : EPAR - Procedural steps taken and scientific information after authorisation

July 12, 2021

CHANGES_SINCE_INITIAL_AUTHORISATION

Orladeyo : EPAR - Public assessment report

June 1, 2021

INITIAL_MARKETING_AUTHORISATION_DOCUMENTS

CHMP summary of positive opinion for Orladeyo

February 26, 2021

INITIAL_MARKETING_AUTHORISATION_DOCUMENTS

Orladeyo : Orphan designation withdrawal assessment report (initial authorisation)

June 1, 2021

INITIAL_MARKETING_AUTHORISATION_DOCUMENTS

Orladeyo-H-C-5138-P46-004 : EPAR - Assessment report

February 13, 2023

CHANGES_SINCE_INITIAL_AUTHORISATION

Orladeyo-H-C-5138-P46-005 : EPAR - Assessment report

July 12, 2023

CHANGES_SINCE_INITIAL_AUTHORISATION

Orladeyo : EPAR – Medicine overview

June 1, 2021

OVERVIEW_DOCUMENT

Orladeyo : EPAR - Risk-management-plan summary

June 1, 2021

RISK_MANAGEMENT_PLAN_SUMMARY

Orladeyo : EPAR - All authorised presentations

June 1, 2021

AUTHORISED_PRESENTATIONS

Orladeyo : Orphan designation withdrawal assessment report (initial authorisation)

June 1, 2021

CHANGES_SINCE_INITIAL_AUTHORISATION

Orladeyo : EPAR - Public assessment report

June 1, 2021

CHANGES_SINCE_INITIAL_AUTHORISATION

CHMP summary of positive opinion for Orladeyo

February 26, 2021

CHANGES_SINCE_INITIAL_AUTHORISATION

Orladeyo-H-C-PSUSA-00010930-202312 : EPAR - Scientific conclusions and grounds for the variation to the terms of the marketing authorisation

September 23, 2024

CHANGES_SINCE_INITIAL_AUTHORISATION

Overview Q&A (7)

Question

How is Orladeyo used?

Answer

Orladeyo is available as capsules and can only be obtained with a prescription. The recommended dose is one 150-mg capsule daily taken with food.

For more information about using Orladeyo, see the package leaflet or contact your healthcare provider.

Question

How does Orladeyo work?

Answer

The active substance in Orladeyo, berotralstat, works by blocking the activity of a protein called kallikrein. In patients with angioedema, overactive kallikrein leads to raised levels of another protein, bradykinin. Bradykinin is involved in a process that causes blood vessels to widen and become leaky. resulting in the swelling and inflammation of angioedema attacks. Blocking the activity of kallikrein reduces the number of these attacks.

Question

What benefits of Orladeyo have been shown in studies?

Answer

Orladeyo was effective at preventing attacks of angioedema in one main study of 121 patients with hereditary angioedema. Patients taking 150 mg Orladeyo every day for 24 weeks had an average of 1.3 attacks per month, compared with 2.4 attacks per month in patients taking placebo (a dummy treatment).

Question

What are the risks associated with Orladeyo?

Answer

The most common side effects with Orladeyo (which may affect more than 1 in 10 people) are headache, abdominal (belly) pain and diarrhoea. Abdominal pain and diarrhoea, which can occur about 1 to 3 months after starting Orladeyo, get better as treatment continues.

For the full list of side effects and restrictions with Orladeyo, see the package leaflet.

Question

Why is Orladeyo authorised in the EU?

Answer

Orladeyo is effective at reducing the number of angioedema attacks, including life-threatening throat swelling. The medicine can be taken by mouth and its side effects were manageable. The European Medicines Agency therefore decided that Orladeyo’s benefits are greater than its risks and it can be authorised for use in the EU.

Question

What measures are being taken to ensure the safe and effective use of Orladeyo?

Answer

Recommendations and precautions to be followed by healthcare professionals and patients for the safe and effective use of Orladeyo have been included in the summary of product characteristics and the package leaflet.

As for all medicines, data on the use of Orladeyo are continuously monitored. Side effects reported with Orladeyo are carefully evaluated and any necessary action taken to protect patients.

Question

Other information about Orladeyo

Answer

Orladeyo received a marketing authorisation valid throughout the EU on 30 April 2021.

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