January 2025 decisions news release
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: “The committee is pleased to be able to accept these new medicines for use by NHSScotland.
“We heard powerful testimony from patient groups about the impact of Duchenne muscular dystrophy on patients and families. Vamorolone will provide an alternative to corticosteroids, providing choice for clinicians and patients.
“Patient group representatives shared the substantial additional impact that facial angiofibromas can have on patients with tuberous sclerosis complex, and the expected benefits that sirolimus, as the first licensed topical gel, could bring.”