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SOLIRIS

Opinions on drugs - Posted on Sep 23 2020

Reason for request

New indication

Key points

Favourable opinion for reimbursement in the treatment of neuromyelitis optica spectrum disorder (NMOSD) in adult patients who are anti-aquaporin-4 (AQP4) antibody-positive with a relapsing course of the disease (2 relapses in the past year or 3 relapses in the past two years, including one in the past year), and not responding to background immunosuppressant therapy (rituximab, azathioprine, mycophenolate mofetil).

Unfavourable opinion for reimbursement in the other clinical situations.

What therapeutic improvement ?

Therapeutic improvement in management of the disease.

Role in the care pathway ?

The treatment of neuromyelitis optica spectrum disorder (NMOSD) is currently based on open-label studies, expert opinions and clinical experience. The background therapies used in practice (off-label) are immunosuppressant treatments, in particular rituximab, azathioprine and mycophenolate mofetil. At present, SOLIRIS (eculizumab) is the only treatment for NMOSD with a marketing authorisation.

Role of the medicinal product in the care pathway

Given the superiority of eculizumab demonstrated in a double-blind, placebo-controlled study in the majority of patients receiving immunosuppressant therapy and in the absence of comparative data versus the immunosuppressant therapies used in practice (off-label), SOLIRIS (eculizumab) is a background therapy for neuromyelitis optica spectrum disorder (NMOSD) in adult patients who are anti-aquaporin-4 (AQP4) antibody-positive with a relapsing course of the disease (2 relapses in the past year or 3 relapses in the past two years, including one in the past year), and not responding to background immunosuppressant therapy (rituximab, azathioprine, mycophenolate mofetil).

Special recommendations

The Committee recommends that treatment with SOLIRIS (eculizumab) be administered in a multiple sclerosis resource and expertise centre or in a centre specialising in rare inflammatory brain and spinal cord diseases, with prescription restricted to neurologists in the context of a multidisciplinary review meeting justified in view of the risk of using SOLIRIS (eculizumab) outside the reimbursement scope defined by the Committee on the basis of available efficacy and safety data, particularly first-line use or use in patients who do not have anti-AQP4 antibodies (off-label).

 

 

 


Clinical Benefit

Substantial

The Committee deems that the clinical benefit of SOLIRIS (eculizumab) is :

  • substantial in the treatment of neuromyelitis optica spectrum disorder (NMOSD) in adult patients who are anti-aquaporin-4 (AQP4) antibody-positive with a relapsing course of the disease (2 relapses in the past year or 3 relapses in the past two years, including one in the past year), and not responding to background immunosuppressant therapy (rituximab, azathioprine, mycophenolate mofetil),

 

Insufficient

The Committee deems that the clinical benefit of SOLIRIS (eculizumab) is :

  • insufficient in other clinical situations.

 

 


Clinical Added Value

moderate

Considering :

  • demonstration of the superiority of eculizumab versus placebo in the treatment of neuromyelitis optica spectrum disorder (NMOSD) in adult patients who are anti-aquaporin-4 (AQP4) antibody-positive with a relapsing course of the disease, in terms of the time to onset and reduction in the annual frequency of relapses, without, however, a demonstrated benefit on disability and quality of life of patients,
  • the clinical relevance of these endpoints,
  • the medical need partially met by immunosuppressant therapies used off-label on the basis of expert consensus,

and despite remaining uncertainties concerning the efficacy and long-term safety, as well as the optimal treatment duration and use strategy in the absence of comparison with the background therapies used in clinical practice, in particular rituximab,

the Transparency Committee considers that SOLIRIS (eculizumab) provides a moderate clinical added value (CAV III) in the treatment of NMOSD in adult patients who are anti-aquaporin-4 (AQP4) antibody-positive with a relapsing course of the disease (2 relapses in the past year or 3 relapses in the past two years, including one in the past year), and not responding to background immunosuppressant therapy (rituximab, azathioprine, mycophenolate mofetil).

 


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