Arikayce liposomal

Authorised

This medicine is authorised for use in the European Union

amikacin

Overview

Arikayce liposomal is an antibiotic for treating adults with a lung infection caused by Mycobacterium avium complex (MAC), a group of bacteria commonly found in the environment, such as in soil and water. It is used in patients with limited treatment options who do not have cystic fibrosis.

Lung infection caused by MAC is rare, and Arikayce liposomal was designated an ‘orphan medicine’ (a medicine used in rare diseases) on 8 April 2014.

Arikayce liposomal contains the active substance amikacin.

How is Arikayce liposomal used?

Amikacin is an antibiotic that belongs to the group 'aminoglycosides'. It works by disrupting the production of proteins that bacteria need to build their cell walls, thereby damaging the bacteria and eventually killing them. In this medicine, amikacin is contained in tiny fat particles known as liposomes, which allows the medicine to remain in the lung for longer.

How does Arikayce liposomal work?

Amikacin is an antibiotic that belongs to the group 'aminoglycosides'. It works by disrupting the production of proteins that bacteria need to build their cell walls, thereby damaging the bacteria and eventually killing them. In this medicine, amikacin is contained in tiny fat particles known as liposomes, which allows the medicine to remain in the lung for longer.

What benefits of Arikayce liposomal have been shown in studies?

A main study in 336 patients showed that Arikayce liposomal is effective at clearing lung infections caused by MAC. After 6 months of treatment, 29% of patients on standard care who used Arikayce liposomal tested negative for the infection, compared with 9% of patients on standard care alone. In addition, 3 months after stopping treatment, 55% of patients whose infections cleared with Arikayce liposomal continued to test negative. No patient who received standard care tested negative 3 months after stopping treatment.

What are the risks associated with Arikayce liposomal?

The most common side effects with Arikayce liposomal affect the lungs and airways: dysphonia (changes to the voice), cough, dyspnoea (difficulty breathing) and haemoptysis (coughing up blood) may affect more than 1 person in 10.

Other common side effects include pain in the mouth or throat, tiredness, diarrhoea, worsening of bronchiectasis (weakened, scarred airways and build-up of mucus) due to infection, nausea (feeling sick) and bronchospasm.

Arikayce liposomal must not be used in patients who are hypersensitive (allergic) to soya, to any aminoglycoside antibiotic or to any ingredient of this medicine. It must also not be used together with another aminoglycoside or in patients with severely impaired kidneys.

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

Why is Arikayce liposomal authorised in the EU?

The main study showed that Arikayce liposomal can clear MAC lung infection in some patients. Although the effect of Arikayce liposomal is modest, the medicine could benefit patients who have few treatment options. In terms of its safety, Arikayce liposomal can cause significant side effects, particularly during the first months of treatment. The Agency therefore recommended restricting the medicine to patients with limited treatment options.

The Agency concluded that the benefits of Arikayce liposomal outweigh its risks and that it can be authorised in the EU.

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

The company will provide patients taking the medicine with an alert card to inform them of the risk of allergic alveolitis (inflammation in the lungs due to an allergy).

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

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

Other information about Arikayce liposomal

Arikayce liposomal received a marketing authorisation valid throughout the EU on 27 October 2020.

Arikayce liposomal : EPAR - Medicine overview

Reference Number: EMA/446084/2020

Arikayce liposomal : EPAR - Risk management plan

Product information

Arikayce liposomal : Product information

Arikayce liposomal : EPAR - All authorised presentations

Product details

• Name of medicine:
  Arikayce liposomal
• Active substance:
  amikacin sulfate
• International non-proprietary name (INN) or common name:
  amikacin
• Therapeutic area (MeSH):
  Respiratory Tract Infections
• Anatomical therapeutic chemical (ATC) code:
  J01GB06

Pharmacotherapeutic group

Antibacterials for systemic use

Therapeutic indication

Arikayce liposomal is indicated for the treatment of non-tuberculous mycobacterial (NTM) lung infections caused by Mycobacterium avium Complex (MAC) in adults with limited treatment options who do not have cystic fibrosis.

Authorisation details

• EMA product number:
  EMEA/H/C/005264
• 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:
  Insmed Netherlands B.V.

  Stadsplateau 7; 3521 AZ Utrecht; Netherlands

• Opinion adopted:
  23/07/2020
• Marketing authorisation issued:
  27/10/2020
• Revision:
  4

Assessment history

Changes since initial authorisation of medicine

Arikayce liposomal : Procedural steps taken and scientific information after authorisation

Arikayce liposomal : Procedural steps taken and scientific information after authorisation (archive)

Arikayce liposomal-H-C-PSUSA-00010882-202209 : EPAR - Scientific conclusions and grounds recommending the variation to the terms of the marketing authorisation

Reference Number: EMA/155354/2023

Initial marketing authorisation documents

Arikayce liposomal : Orphan designation assessment report (initial authorisation)

Adopted Reference Number: EMADOC-1700519818-518626

Arikayce liposomal : EPAR - Public assessment report

Adopted Reference Number: EMA/473660/2020

CHMP summary of positive opinion for Arikayce liposomal

Adopted Reference Number: EMA/CHMP/257409/2020 Corr.