What has SMC said?
The Scottish Medicines Consortium (SMC) has accepted pegunigalsidase alfa, for the long-term treatment of adults with Fabry disease (a rare inherited condition where patients do not have enough of an enzyme called alpha galactosidase).
SMC has accepted pegunigalsidase alfa for restricted use. The restriction means that it is accepted for use to treat adults with Fabry disease who are experiencing symptoms and who would usually be offered an enzyme replacement therapy.
This document summarises the SMC decision and what it means for patients.
You can find more detailed information about the SMC assessment of pegunigalsidase alfa by looking at the SMC detailed advice document (SMC2665).
What does SMC's decision mean for patients?
Pegunigalsidase alfa for use as described above is available for prescribing on the NHS in Scotland. Your healthcare professional can discuss with you if it is the right treatment for you.
You can find more information about making decisions about your treatment in this booklet called: Medicines in Scotland: What’s the right treatment for me?
More about SMC's decision
SMC’s decision takes into account a confidential discount offered by the pharmaceutical company.
How does SMC make its decision?
SMC carefully considers every new medicine to make sure it benefits patients and is likely to be a good use of NHS resources.
To do this SMC studies the following:
- Evidence from the company about how well the medicine works compared with current treatments available in Scotland, in relation to how much they will cost to buy and use to treat patients.
- Information from patient groups about the potential impact of the medicine on patients and carers.
- Advice from healthcare professionals about any benefits of the new medicine compared to current treatment, along with how the new medicine is likely to be used.
When SMC assesses a medicine it takes account of the needs of all patients in NHSScotland, not only those who may be treated with the medicine.
You can find out more about how SMC decides here: htps://www.scottishmedicines.org.uk/how-we-decide/
More information and support
The organisation below can give more information and support for people with Fabry disease and their families. SMC is not responsible for the content of any information provided by external organisations.
MPS Society
https://mpssociety.org.uk
0345 389 9901
You can find out more about pegunigalsidase alfa (brand name: Elfabrio®) in the Patient Information Leaflet (PIL) by searching for the medicine name on the Medicines and Healthcare products Regulatory Agency (MHRA) website.
https://products.mhra.gov.uk/