following a resubmission assessed under the orphan medicine process:

mercaptamine (Procysbi®) is not recommended for use within NHSScotland.

Indication under review: For the treatment of proven nephropathic cystinosis.

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.

The submitting company’s justification of the treatment’s cost in relation to its health benefits was not sufficient and in addition the company did not present a sufficiently robust economic analysis to gain acceptance by SMC.

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

The submitting company has indicated their intention to make a resubmission.

Medicine details

Medicine name:
mercaptamine (Procysbi)

For the treatment of proven nephropathic cystinosis.

Pharmaceutical company
Chiesi Limited
BNF chapter
Nutrition and blood
Submission type
Not recommended
Date advice published
13 September 2021