/Human medicines European public assessment report (EPAR): Waylivra, volanesorsen, Date of authorisation: 03/05/2019, Revision: 8, Status: Authorised

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Human medicines European public assessment report (EPAR): Waylivra, volanesorsen, Date of authorisation: 03/05/2019, Revision: 8, Status: Authorised

EMA

2026/03/23

Human medicines European public assessment report (EPAR): Waylivra, volanesorsen, Date of authorisation: 03/05/2019, Revision: 8, Status: Authorised

Application under evaluation
CHMP opinion
European Commission decision

Overview

Waylivra is a medicine used to treat familial chylomicronaemia syndrome (FCS), a genetic condition that gives rise to high levels of fats called triglycerides in the blood. Excess fat builds up in various parts of the body and leads to symptoms including abdominal pain (belly ache), deposits of fat under the skin and pancreatitis (inflammation of the pancreas).

Waylivra, along with a low fat diet, is used to reduce triglyceride blood levels in patients with FCS that has been confirmed by genetic testing. It is only given to patients in whom other medicines to reduce triglycerides have not worked and who are at high risk of developing pancreatitis.

FCS is rare, and Waylivra was designated an ‘orphan medicine’ (a medicine used in rare diseases) on 19 February 2014.

Waylivra contains the active substance volanesorsen.

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How is Waylivra used?

Waylivra is available as a solution for injection under the skin, which patients or their carers can inject themselves once they have been trained.

At the start of treatment, Waylivra is given once a week. After 3 months, patients who have had a sufficient reduction in the level of triglycerides can continue to receive the medicine once every 2 weeks. The frequency of injections is adjusted again after 6 and 9 months depending on how well the medicine is working.

Because Waylivra can reduce blood levels of platelets (components that help the blood to clot), patients’ blood platelet levels should be checked regularly, in addition to regular checks of triglycerides to see how well the medicine is working. Depending on the results, frequency of injections may be adjusted, or treatment paused or discontinued.

Waylivra can only be obtained with a prescription and treatment should be supervised by a doctor who has experience in treating FCS. For more information about using Waylivra, see the package leaflet or contact your doctor or pharmacist.

How does Waylivra work?

The active substance in Waylivra, volanesorsen, is an ‘antisense oligonucleotide,’ a very short piece of synthetic RNA (a type of genetic material). It has been designed to block the production of a protein that slows down the breakdown of fats called apolipoprotein C-III. By blocking the production of this protein, the medicine reduces the level of triglycerides in the blood and, as a result, fat accumulation in the body, which is expected to reduce the risk of pancreatitis.

What benefits of Waylivra have been shown in studies?

Waylivra was effective in reducing triglycerides in the blood in a study of 67 patients with FCS. After 3 months, patients given Waylivra had an average 77% reduction in the level of triglycerides compared with an average 18% reduction in patients given placebo (a dummy treatment). All patients in the study were on a low fat diet in addition to receiving Waylivra or placebo.

What are the risks associated with Waylivra?

The most common side effects with Waylivra are reduced platelet levels (which may affect 4 in 10 people) and reactions at the site of the injection such as pain, swelling, itching, or bruising (which may affect 8 in 10 people).

Waylivra must not be used in patients who have long-term or unexplained thrombocytopenia (low blood platelet levels).

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

Why is Waylivra authorised in the EU?

Waylivra effectively reduces triglyceride levels and is expected to reduce the risk of pancreatitis in patients with FCS. The main risk with this medicine is thrombocytopenia which, when severe, can lead to bleeding. Patients must therefore be monitored closely and the frequency of injections adjusted if needed.

The European Medicines Agency decided that, for patients at high risk of pancreatitis and for whom other medicines and a low-fat diet have not worked well enough, Waylivra’s benefits are greater than its risks and that it can be authorised for use in the EU.

Waylivra has been given ‘conditional authorisation’. This means that there is more evidence to come about the medicine, which the company is required to provide. Every year, the Agency will review any new information that becomes available and this overview will be updated as necessary.

What information is still awaited for Waylivra?

Since Waylivra has been given conditional authorisation, the company that markets Waylivra will provide results of a study based on a registry of patients to investigate how blood checks and adjustments to frequency of injections are carried out in practice and how well they work to prevent thrombocytopenia and bleeding.

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

The company that markets Waylivra will provide educational materials for patients and healthcare professionals with information on the risk of thrombocytopenia and bleeding with treatment and the importance of regularly checking platelet levels in patients.

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

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

Other information about Waylivra

Waylivra received a conditional marketing authorisation valid throughout the EU on 3 May 2019.

Waylivra : EPAR - Medicine overview

Reference Number:

EMA/161242/2019

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Waylivra : EPAR - Risk management plan

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Product information

Waylivra : EPAR - Product Information

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Latest procedure affecting product information:

VR/0000280220

11/07/2025

This medicine’s product information is available in all official EU languages. Select 'available languages' to access the language you need.

Product information documents contain:

summary of product characteristics (annex I);
manufacturing authorisation holder responsible for batch release (annex IIA);
conditions of the marketing authorisation (annex IIB);
labelling (annex IIIA);
package leaflet (annex IIIB).

Waylivra : EPAR - All Authorised presentations

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Product details

Name of medicine

Waylivra

Active substance

volanesorsen sodium

International non-proprietary name (INN) or common name

volanesorsen

Therapeutic area (MeSH)

Hyperlipoproteinemia Type I

Anatomical therapeutic chemical (ATC) code

C10AX18

Pharmacotherapeutic group

Other lipid modifying agents

Therapeutic indication

Waylivra is indicated as an adjunct to diet in adult patients with genetically confirmed familial chylomicronemia syndrome (FCS) and at high risk for pancreatitis, in whom response to diet and triglyceride lowering therapy has been inadequate.

Authorisation details

EMA product number

EMEA/H/C/004538

Additional monitoring

This medicine is under additional monitoring, meaning that it is monitored even more intensively than other medicines. For more information, see Medicines under additional monitoring.

Conditional approval

This medicine received a conditional marketing authorisation. This was granted in the interest of public health because the medicine addresses an unmet medical need and the benefit of immediate availability outweighs the risk from less comprehensive data than normally required. For more information, see Conditional marketing authorisation.

Orphan

This medicine was designated an orphan medicine. This means that it was developed for use against a rare, life-threatening or chronically debilitating condition or, for economic reasons, it would be unlikely to have been developed without incentives. For more information, see Orphan designation.

Marketing authorisation holder

Akcea Therapeutics Ireland Limited

Akcea Therapeutics Ireland Limited St. James House 72 Adelaide Road, Dublin 2 D02 Y017 Ireland

Opinion adopted

28/02/2019

Marketing authorisation issued

03/05/2019

Revision

Assessment history

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Changes since initial authorisation of medicine

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

English (EN) (149.08 KB - PDF)

First published:

14/07/2025

Last updated:

23/03/2026

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Waylivra : EPAR - Procedural steps taken and scientific information after authorisation (archive)

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First published:

20/08/2019

Last updated:

14/07/2025

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Initial marketing authorisation documents

Waylivra : Orphan maintenance assessment report (initial authorisation)

Adopted

Reference Number:

EMA/202821/2019

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First published:

20/05/2019

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Waylivra : EPAR - Public assessment report

Adopted

Reference Number:

EMA/180717/2019

English (EN) (2.48 MB - PDF)

First published:

20/05/2019

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CHMP summary of positive opinion for Waylivra

Adopted

Reference Number:

EMA/CHMP/130311/2019

English (EN) (69.15 KB - PDF)

First published:

01/03/2019

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News on Waylivra

Meeting highlights from the Committee for Medicinal Products for Human Use (CHMP) 25-28 February 2019

01/03/2019

First treatment for rare disease characterised by high levels of triglycerides in blood

01/03/2019

More information on Waylivra

Topics

This page was last updated on

23/03/2026

Summary

Human medicines European public assessment report (EPAR): Waylivra, volanesorsen, Date of authorisation: 03/05/2019, Revision: 8, Status: Authorised