ADEMPAS (riociguat) - PAH in children and adolescents

The Committee deems that the clinical benefit of ADEMPAS (riociguat) film-coated tabletsis moderate in the treatment of PAH in children and adolescents less than 18 years of age and body weight ≥ 50 kg with WHO Functional Class (FC) II to III in combination with endothelin receptor antagonists.

Considering:

• the results observed in the paediatric population compared to those in adults in a study assessing the pharmacokinetics of riociguat, suggesting the transposability to the paediatric population of the clinical results demonstrated in adults;
• the lack of evidence of clinical efficacy of ADEMPAS (riociguat), particularly in terms of morbidity and mortality, in children and adolescents with WHO Functional Class (FC) II to III PAH less than 18 years of age and body weight ≥ 50 kg, due to the absence of comparative data;
• the safety profile that appears to be similar to that of adults;

the Committee deems that ADEMPAS (riociguat) film-coated tablets provide no clinical added value (CAV V) compared to the relevant comparators (see paragraph 5.2) in the current care pathway in children and adolescents with WHO Functional Class (FC) II to III pulmonary arterial hypertension less than 18 years of age and body weight ≥ 50 kg.