June 2023 decisions news release
The Scottish Medicines Consortium (SMC), which advises on newly-licensed medicines for use by NHSScotland, has today (Monday, June 12) published advice on six medicines.
An initial assessment report has been published for metreleptin (Myalepta®) for the treatment of adults and children over two with lipodystrophy, a very rare condition in which the body is unable to maintain healthy fat tissue resulting in severe abnormalities and reduced life expectancy.
Metreleptin has been assessed through the ultra-orphan pathway for medicines aimed at extremely rare conditions. It will be available for three years while further information is gathered to help address key uncertainties with the clinical and cost effectiveness assessments. Following this, SMC will review the evidence and make a decision on routine availability in NHSScotland.
Pembrolizumab (Keytruda®) was accepted for use in patients with locally advanced or early stage triple negative breast cancer at high risk of recurrence, as part of treatment that includes chemotherapy and surgery.
Nivolumab (Opdivo®) was accepted as a first-line treatment, alongside chemotherapy, for cancer of the oesophagus. This cancer forms in the cells lining the oesophagus.
Polatuzumab vedotin (Polivy®) was accepted for the first-line treatment of adults with diffuse large B-cell lymphoma, a type of blood cancer. It is given as part of a chemotherapy regimen.
Treosulfan (Trecondi®) was accepted as part of treatment given to patients before they undergo a donor stem cell transplant.
Upadacitinib (Rinvoq®) was accepted as a treatment option for people with moderate to severe Crohn’s disease who have not had benefit from other standard treatments.
SMC Chair, Dr Scott Muir, said: “Liposystrophy is a debilitating condition that can result in high morbidity with multi-organ damage of the liver, kidneys and pancreas, poor quality of life and premature death. There are no other medicines currently licensed for the treatment of this condition.
“The Scottish Government will announce when metreleptin will be available for prescribing. The cost of metreleptin is currently very high; availability via the ultra-orphan pathway will allow the company more time to gather data in order to address key uncertainties identified by the committee. Thereafter SMC will reassess the case to ensure that this medicine is a good use of NHS resources.
“We know that triple negative breast cancer can be an aggressive disease with a high risk of occurrence. Adding pembrolizumab to current treatment has been shown to improve response rates which will be welcomed by clinicians and patients in Scotland.”