riociguat (Adempas)

following a full submission considered under the orphan process

riociguat (Adempas®) is accepted for restricted use within NHS Scotland.

Indication under review: Pulmonary arterial hypertension (PAH): as monotherapy or in combination with endothelin receptor antagonists, for the treatment of adult patients with PAH with World Health Organisation Functional Class (WHO FC) II to III to improve exercise capacity. Efficacy has been shown in a PAH population including aetiologies of idiopathic or heritable PAH or PAH associated with connective tissue disease.

SMC restriction: for use as a PAH-specific monotherapy as an alternative treatment option to endothelin receptor antagonist (ERA) monotherapy in adult patients with PAH of WHO FC II to III. It is restricted to initiation and prescribing by specialists in the Scottish Pulmonary Vascular Unit or by similar specialists.

Riociguat demonstrated significant improvement compared with placebo in exercise capacity, in terms of six-minute walking distance, in patients with symptomatic PAH in a phase III study.

This advice takes account of the benefits of a Patient Access Scheme (PAS) that improves the cost-effectiveness of riociguat. This advice is contingent upon the continuing availability of the patient access scheme in NHS Scotland or a list price that is equivalent or lower.

This advice takes account of the views from a Patient and Clinician Engagement (PACE) meeting.