Media release: SMC decisions January 2025

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The Scottish Medicines Consortium (SMC), which advises on newly licensed medicines for use by NHSScotland, has today (Monday 13 January) published advice on nine medicines.

Vamorolone (Agamree®) was accepted as a treatment for Duchenne muscular dystrophy in patients aged four years and older. Duchenne muscular dystrophy is a rare genetic disorder that causes muscle weakness and loss of function.

Sirolimus gel (Hyftor®) was accepted as a treatment for facial angiofibroma, a non-cancerous skin tumour on the face that is caused by a rare genetic condition called tuberous sclerosis complex. Sirolimus is used in patients aged six years and older.

Three different medicines were accepted to treat anaemia in patients with paroxysmal nocturnal haemoglobinuria, a rare condition where the body’s immune system is overactive and breaks down too many blood cells. Danicopan (Voydeya®) is used in adults, together with other medicines. Iptacopan (Fabhalta®) is used in adults on its own and crovalimab (Piasky®) is used in patients who are 12 years or over.

An ultra-orphan initial assessment report has been published for fosdenopterin (Nulibry®) a treatment for molybdenum cofactor deficiency Type A, a very rare genetic condition which causes brain damage. The Scottish Government will confirm when fosdenopterin is available for prescribing and further evidence will be gathered. Following this, SMC will review it and decide on routine availability in NHSScotland.

Relugolix/estradiol/norethisterone  (Ryeqo®) was accepted to treat endometriosis in adult women of reproductive age.

Risankizumab (Skyrizi®) was accepted for the treatment of adults with moderate to severe ulcerative colitis.

Ublituximab (Briumvi®) was accepted for the treatment of adults with relapsing forms of multiple sclerosis.

SMC Chair Dr Scott Muir said:

Ends

Notes to editor

1. The Scottish Medicines Consortium (SMC) is the national source of advice on the clinical and cost-effectiveness of all new medicines for NHSScotland. Our aim is to ensure that people in Scotland have timely access to beneficial new medicines.
2. We are part of Healthcare Improvement Scotland. Our committee is made up of clinicians, pharmacists, NHS board representatives, the pharmaceutical industry and the public. Most of the clinicians have a direct role in patient care, while our three volunteer public partners ensure the views of the Scottish public are taken into account during decision making. This wide mixture of backgrounds ensures decisions are made from a broad perspective.
3. Members of the SMC Committee make their decisions based on a broad range of evidence in order to help health professionals deliver the best possible care within the finite resources available. They consider detailed evidence presented by pharmaceutical companies, patient groups and clinicians in order to decide which medicines provide value for money for NHSScotland.
4. When we talk about value for money or cost effectiveness, this does not just mean how much a medicine costs to buy, but the wider costs and benefits associated with it being regularly used to treat those with a particular condition. The NHS does not have unlimited resources and if we accept a medicine where the benefits are not clear it may mean other patients lose out.
5. When considering a new medicine we look at:

• how well the medicine works
• which patients with the particular condition would benefit from it
• whether it is as good as or better than the medicines already used to treat the particular condition, and
• whether it is good value for money.

For more information on how we make our decisions, go to the SMC website, https://www.scottishmedicines.org.uk/how-we-decide/

1. Changes to the way we assess medicines for end of life and rare conditions (https://www.scottishmedicines.org.uk/how-we-decide/pace/) have allowed our committee additional flexibility in decision making. These changes include the option of a Patient and Clinician Engagement (PACE) meeting which gives patient groups and clinicians the opportunity to provide additional evidence to the committee.
2. A new approach to the assessment of medicines which treat extremely rare conditions has been introduced (https://www.scottishmedicines.org.uk/how-we-decide/ultra-orphan-medicines-for-extremely-rare-conditions/) in line with the Scottish Government announcement in June 2018 on a new pathway (https://news.gov.scot/news/treatments-for-rare-conditions)
3. SMC can make the following decisions on a medicine:

• accepted
• accepted with a restriction(s) (for example, the medicine can only be accepted in a particular group of patients with the condition. This typically occurs because the company has requested this explicitly in the submission)
• accepted on an interim basis, or
• not recommended

SMC can accept some medicines on an interim basis subject to ongoing evaluation and reassessment. You can find out more about this on our website https://www.scottishmedicines.org.uk/how-we-decide/interim-acceptance-decision-option/

SMC may be unable to accept a medicine for use in Scotland if the committee is not satisfied that the benefits of the medicine offer value for money for patients and the NHS or where there is a high degree of uncertainty about the clinical benefits of the medicine. You can find out more about the reasons we may not recommend medicines on our website: https://www.scottishmedicines.org.uk/how-we-decide/

1. For medicines that have not been recommended, SMC welcomes a resubmission from the company at any time with new clinical and/or economic evidence.
2. When a medicine has not been recommended by SMC, all NHS boards have procedures in place to consider individual requests when a doctor feels the medicine would be right for a particular patient.
3. For further information and to view the complete advice for the medicines detailed above, visit our website at: www.scottishmedicines.org.uk.
4. For further information on ILAP, please visit the MHRA website https://www.gov.uk/guidance/innovative-licensing-and-access-pathway