July 2024 decisions news release
The Scottish Medicines Consortium (SMC), which advises on newly licensed medicines for use by NHSScotland, has today (Monday, July 8) published advice on eight medicines.
Voretigene neparvovec (Luxturna®) was accepted for the treatment of adults and children with an extremely rare inherited eye condition called inherited retinal dystrophy. It has been available for use as part of the ultra-orphan pathway, since 2020, while further evidence on its effectiveness was collected. Following reassessment, it has now been accepted by SMC for routine use.
An ultra-orphan initial assessment report has been published for birch bark extract (Filsuvez®) a treatment for epidermolysis bullosa (EB), a rare inherited skin condition that makes the skin very fragile and causes severe blistering and scarring. The Scottish Government will confirm when it is available for prescribing and further evidence will be gathered. Following this SMC will review it and decide on routine availability in NHSScotland.
Pegunigalsidase alfa (Elfabrio®) was accepted as a treatment for adults with Fabry disease, a rare inherited condition that can lead to kidney failure and heart problems.
Empagliflozin (Jardiance®) was accepted for the treatment of adults with chronic kidney disease, a long-term condition where the kidneys don’t work as well as they should.
Nivolumab/relatlimab (Opdualag®) was accepted for the treatment of advanced melanoma, a type of skin cancer, in patients aged 12 years and older.
Follitropin delta (Rekovelle®) was accepted for ovarian stimulation in women having fertility treatments such as in vitro fertilisation (IVF) or intracytoplasmic sperm injection (ICSI).
Pembrolizumab (Keytruda®) used together with chemotherapy was accepted as a first-line treatment for adults with HER2-negative advanced gastric or gastro-oesophageal junction cancer. It was not recommended for those with HER2-positive disease, when used together with chemotherapy and trastuzumab.
SMC Chair Dr Scott Muir said: “The committee is pleased to be able to accept these new medicines for use by NHSScotland.
“Through the SMC Patient and Clinician Engagement process, the committee heard about the devastating and life-changing impact of progressive vision loss caused by inherited retinal dystrophy. From the additional evidence provided by the company, the committee was satisfied that voretigene neparvovec has the potential to improve the lives of patients and their families and has now been accepted for routine use by SMC.
“The committee was only able to accept pembrolizumab for advanced gastric or gastro-oesophageal junction cancer in patients with HER2-negative disease as the evidence provided by the company for pembrolizumab when used to treat HER2-positive disease was not strong enough. We would welcome a resubmission addressing the uncertainties that we have raised.”