mercaptamine (Procysbi)

following a third resubmission assessed under the orphan equivalent medicine process:

mercaptamine (Procysbi®) is accepted for use within NHSScotland.

Indication under review: treatment of proven nephropathic cystinosis. Cysteamine reduces cystine accumulation in some cells (e.g. leukocytes, muscle and liver cells) of nephropathic cystinosis patients and, when treatment is started early, it delays the development of renal failure.

A phase III, open-label, crossover study demonstrated that extended-release mercaptamine (Procysbi®) was non-inferior to immediate-release mercaptamine in control of white blood cell cystine levels in patients with nephropathic cystinosis who were previously controlled on mercaptamine therapy.

This advice applies only in the context of an approved NHSScotland Patient Access Scheme (PAS) arrangement delivering the cost-effectiveness results upon which the decision was based, or a PAS/ list price that is equivalent or lower.

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