following a full submission assessed under the orphan medicine process:
sirolimus (Hyftor®) is accepted for use within NHSScotland.
Indication under review: for the treatment of facial angiofibroma associated with tuberous sclerosis complex in adults and paediatric patients aged 6 years and older.
In a randomised phase III study, sirolimus gel demonstrated a statistically significant improvement in facial angiofibromas at week 12 compared with placebo.
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.
Medicine details
- Medicine name:
- sirolimus (Hyftor)
- SMC ID:
- SMC2710
- Indication:
For the treatment of adult and paediatric patients aged 6 and above with facial angiofibroma (facial AF) associated with tuberous sclerosis complex (TSC).
- Pharmaceutical company
- Plusultra pharma
- BNF chapter
- Skin
- Submission type
- Full
- Status
- Accepted
- Date advice published
- 13 January 2025