Amvuttra | European Medicines Agency (EMA)

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Overview

Amvuttra is a medicine used to treat polyneuropathy (nerve damage) caused by hereditary transthyretin-mediated (hATTR) amyloidosis, a disease in which abnormal proteins called amyloids build up in tissues around the body including around the nerves.

Amvuttra is used in adult patients in the first two stages of the nerve damage (stage 1, when the patient has weakness in the legs but is able to walk unaided, and stage 2, when the patient can still walk but needs help).

hATTR amyloidosis is rare, and Amvuttra was designated an ‘orphan medicine’ (a medicine used in rare diseases) on 25 May 2018. Further information on the orphan designation can be found here.

Amvuttra contains the active substance vutrisiran.

Amvuttra can only be obtained with a prescription and treatment should be started and supervised by a doctor experienced in the treatment of patients with amyloidosis. Treatment should begin as early as possible after diagnosis, to avoid further progression of the disease.

The medicine is available as an injection under the skin (subcutaneous) in the abdomen, thigh, or upper arm. The recommended dose is 25 mg once every 3 months. Patients should also take vitamin A supplements during treatment with Amvuttra.

For patients whose disease progresses to stage 3 polyneuropathy, the doctor may continue treatment if the benefits outweigh the risks.

For more information about using Amvuttra, see the package leaflet or contact your doctor or pharmacist.

In patients with hATTR amyloidosis, a protein called transthyretin which circulates in the blood is defective and breaks easily. The broken protein forms amyloid deposits in tissues and organs around the body, including around nerves, where it interferes with the normal organ function.

The active substance in Amvuttra, vutrisiran, is a ‘small interfering RNA’ (siRNA), a very short piece of synthetic genetic material, that has been designed to attach to and block the genetic material of the cells responsible for producing transthyretin. This reduces production of defective transthyretin, thereby reducing the formation of amyloids and relieving the symptoms of hATTR amyloidosis.

In one main study involving 164 patients with hATTR amyloidosis with stage 1 or 2 nerve damage, Amvuttra was shown effective at slowing down the nerve damage caused by the disease.

The main measure of effectiveness was the change in the patients’ nerve damage, as measured by a standard scale called ‘mNIS+7’, where a decreased score indicates an improvement and an increased score indicates worsening nerve damage. After 18 months of treatment, the mNIS+7 score decreased on average by around 0.5 points with Amvuttra. This was compared with an average increase of 28 points seen with placebo (a dummy treatment) in another study involving 225 patients comparing Onpattro (another hATTR amyloidosis medicine) with placebo.

The study also showed that treatment with Amvuttra was at least as effective as Onpattro at reducing transthyretin levels.

The most common side effects with Amvuttra (which may affect more than 1 in 10 people) are pain in the extremities (arms and legs) and arthralgia (joint pain).

For the full list of side effects of Amvuttra, see the package leaflet.

Amvuttra was shown to be effective at slowing down nerve damage in patients with hATTR amyloidosis with stage 1 or stage 2 nerve damage. Regarding safety, the side effects are considered manageable.

The European Medicines Agency decided that Amvuttra’s benefits are greater than its risks and it can be authorised for use in the EU.

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

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

Amvuttra received a marketing authorisation valid throughout the EU on 15 September 2022.

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First published: 12/10/2022

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English (EN)
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First published: 12/10/2022Last updated: 17/01/2024

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Product information

English (EN)
(657.75 KB – PDF)

First published: 12/10/2022Last updated: 22/01/2025

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Latest procedure affecting product information:
II/0013/G

28/11/2024

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).

English (EN)
(40.18 KB – PDF)

First published: 12/10/2022

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Product details

Name of medicine: Amvuttra
Active substance: vutrisiran sodium
International non-proprietary name (INN) or common name: vutrisiran
Therapeutic area (MeSH): Amyloid Neuropathies, Familial
Anatomical therapeutic chemical (ATC) code: N07XX18

Pharmacotherapeutic group

Other nervous system drugs

Therapeutic indication

Treatment of hereditary transthyretin-mediated amyloidosis (hATTR amyloidosis) in adult patients with stage 1 or stage 2 polyneuropathy.

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