ARIKAYCE LIPOSOMAL (amikacine)

Key points

Favourable opinion for reimbursement in 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.

What therapeutic improvement?

Therapeutic improvement in management of the condition.

Role in the care pathway?

The treatment of lung infections caused by Mycobacterium avium Complex (MAC) is well standardised in the international guidelines and is based on a combination of antibiotics: multiple drug regimen (MDR). This is composed of rifampicin or rifabutin, a macrolide (clarithromycin or azithromycin) and ethambutol continued for 12 months following conversion to negative sputum cultures. The addition of parenteral amikacin is envisaged in cavitary or severe forms and for macrolide-resistant infections.

In the event of non-response (patients without sputum culture conversion after 6 consecutive months of MDR), the use of inhaled liposomal amikacin in conjunction with MDR is recommended.

Role of the medicinal product in the care pathway

Considering demonstration of its microbiological efficacy (durable sputum culture conversion in heavily pretreated patients), the Transparency Committee considers that ARIKAYCE LIPOSOMAL (amikacin) is a salvage therapy, in conjunction with MDR, in the event of failure of oral MDR therapy alone (at least 6 months of treatment), in patients with lung infections caused by MAC sensitive to amikacin, with limited treatment options and who do not have cystic fibrosis. Patients with cystic fibrosis were not included in the pivotal study.

Special recommendations

The Committee highlights the fact that treatment should be initiated and monitored by physicians experienced in the management of these incapacitating and difficult-to-treat infections. In addition, patients should be informed about both the benefits of the treatment and the safety issues, i.e., the poor respiratory tolerance (related to aerosolization), the long-term risks, in particular tinnitus, which can impair quality of life, and ototoxicity, which may be more problematic and irreversible. To limit this risk, approximate cumulative doses with an increased probability of ototoxicity must be determined, particularly when there is a risk of renal impairment.