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mercaptamine (Procysbi)

Advice

following a full submission considered under the ultra-orphan process:

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

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

In a phase III study delayed release mercaptamine (Procysbi®) was non-inferior to another formulation of mercaptamine, in maintaining white blood cell cystine levels at a safe level, 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 clinical or economic analysis to gain acceptance by SMC.

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

Drug Details

Drug Name: mercaptamine (Procysbi)
SMC Drug ID: 1272/17
Manufacturer: Horizon Pharma
Indication: For the treatment of proven nephropathic cystinosis.
BNF Category:
Submission Type: Full submission
Status: Not Recommended
Date Advice Published: 13 November 2017

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