MedPath
EMA Approval

Waylivra

C10AX18

volanesorsen

Other lipid modifying agents

volanesorsen

Hyperlipoproteinemia Type I

Basic Information

EMA regulatory identification and product classification information

EMA Identifiers

ATC CodeC10AX18
EMA European Classification

Overview Summary

Comprehensive product overview and regulatory summary

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.

Authorisations (2)

EMEA/H/C/004538

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

Authorised

May 3, 2019

Orphan MedicineConditional Approval

EMEA/H/C/004538

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

Authorised

May 3, 2019

Active Substances (2)

volanesorsen sodium

Volanesorsen sodium

Documents (12)

Waylivra : Orphan maintenance assessment report (initial authorisation)

May 20, 2019

INITIAL_MARKETING_AUTHORISATION_DOCUMENTS

Waylivra : EPAR - Public assessment report

May 20, 2019

INITIAL_MARKETING_AUTHORISATION_DOCUMENTS

CHMP summary of positive opinion for Waylivra

March 1, 2019

INITIAL_MARKETING_AUTHORISATION_DOCUMENTS

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

July 14, 2025

CHANGES_SINCE_INITIAL_AUTHORISATION

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

August 20, 2019

CHANGES_SINCE_INITIAL_AUTHORISATION

Waylivra : EPAR - Product Information

May 20, 2019

DRUG_PRODUCT_INFORMATION

Waylivra : EPAR - Risk-management-plan summary

May 20, 2019

RISK_MANAGEMENT_PLAN_SUMMARY

Waylivra : Orphan maintenance assessment report (initial authorisation)

May 20, 2019

CHANGES_SINCE_INITIAL_AUTHORISATION

Waylivra : EPAR - Public assessment report

May 20, 2019

CHANGES_SINCE_INITIAL_AUTHORISATION

CHMP summary of positive opinion for Waylivra

March 1, 2019

CHANGES_SINCE_INITIAL_AUTHORISATION

Waylivra : EPAR - All Authorised presentations

May 20, 2019

AUTHORISED_PRESENTATIONS

Waylivra : EPAR - Medicine overview

May 20, 2019

OVERVIEW_DOCUMENT

Overview Q&A (8)

Question

How is Waylivra used?

Answer

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.

Question

How does Waylivra work?

Answer

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.

Question

What benefits of Waylivra have been shown in studies?

Answer

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.

Question

What are the risks associated with Waylivra?

Answer

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.

Question

Why is Waylivra authorised in the EU?

Answer

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.

Question

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

Answer

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.

Question

Other information about Waylivra

Answer

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

Question

What information is still awaited for Waylivra?

Answer

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.

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Waylivra - EMA Approval | MedPath