REBLOZYL (luspatercept) - Myelodysplastic syndromes (MDS)

The clinical benefit of REBLOZYL 25 mg and 75 mg (luspatercept) powder for solution for injection in the context of the indication extension isubstantial only in “the treatment of transfusion-dependent anaemia due to very low, low and intermediate-risk MDS in adults with no prior treatment with erythropoiesis-stimulating agents (ESA) with ring sideroblasts and without 5q deletion”.

Considering:

• available data from the phase 3 study demonstrating the superiority of luspatercept versus epoetin alfa in terms of transfusion independence in the short term (24 weeks),
• the clinical relevance of this criterion and the benefit observed,

and despite:

• the open-label nature of the study,
• the lack of evidence of an impact on quality of life and fatigue, the main symptom responsible for functional disability,
• the safety profile of luspatercept, globally less favourable than that of epoetin alfa,
• the absence of robust comparative data on duration of response and haemoglobin levels
• uncertainties with respect to the maintenance of efficacy in the long term, as well as clinical endpoints of interest, such as patient survival and disease progression,

the Committee deems that REBLOZYL (luspatercept) provides a minor clinical added value (CAV IV) compared to erythropoiesis-stimulating agents (EPO therapy) in the treatment of transfusion-dependent anaemia due to very low, low and intermediate-risk MDS in adults with no prior treatment with erythropoiesis-stimulating agents (ESA) with ring sideroblasts and without 5q deletion.