September 2023 decisions news release
The Scottish Medicines Consortium (SMC), which advises on newly-licensed medicines for use by NHSScotland, has today (Monday, September 11) published advice on seven medicines.
Rimegepant (Vydura®) was accepted for the prevention of migraine in adults who have at least four migraine attacks per month.
Daratumumab (Darzalex®) was accepted as part of a combination treatment, for newly diagnosed myeloma, a rare type of blood cancer. It will be used to treat patients who cannot have a transplant using their own blood producing cells (an autologous stem cell transplant).
Ibrutinib (Imbruvica®) was accepted for use together with another medicine called venetoclax (Venclyxto®), for the treatment of previously untreated chronic lymphocytic leukaemia (CLL), a type of blood cancer.
Vutrisiran (Amvuttra®) was accepted for the treatment of hereditary transthyretin-mediated amyloidosis (hATTR amyloidosis) in adult patients with stage one or two polyneuropathy. hATTR amyloidosis is a very rare condition where abnormal proteins called amyloid build up in the tissues of the body including around the nerves. Patients can suffer heart and neurological problems, among a wide range of symptoms.
Two medicines have been assessed through the ultra-orphan pathway for medicines to treat extremely rare conditions. The medicines, eladocagene exuparvovec (Upstaza®) and olipudase alfa (Xenpozyme®), will be available for three years while further information is gathered. Following this, SMC will review the evidence and make a decision on routine availability in NHSScotland.
Eladocagene exuparvovec (Upstaza®) is the first gene therapy medicine to treat severe aromatic L-amino acid decarboxylase (AADC) deficiency in adults and children. AADC deficiency is a very rare and severely debilitating condition caused by a genetic mutation that affects the nervous system. It causes developmental delays, muscle weakness, movement disorders and intellectual disability.
Olipudase alfa (Xenpozyme®) is used to treat adults and children with a very rare genetic condition called acid sphingomyelinase deficiency (ASMD). ASMD is also known as Niemann-Pick disease. It is the first medicine available to target the underlying cause of this very rare condition that can affect the spleen, lungs, liver and blood.
The committee was unable to accept mosunetuzumab (Lunsumio®) for the treatment of patients with follicular lymphoma, a type of blood cancer. The company’s evidence around the clinical and cost effectiveness of the treatment compared to currently available options was not sufficient.
SMC Chair, Dr Scott Muir, said: “We are very pleased to be able to accept these medicines for use by NHSScotland.
“Rimegepant (Vydura) offers an oral treatment option which may help improve symptoms and quality of life for those affected by migraines.
“We know from the Patient and Clinician Engagement (PACE) meetings that daratumumab and ibrutinib are considered valuable treatment options so we are pleased to be able to accept these medicines for use in people with rare blood cancers.”