Regulator to rule on new drugs for spinal condition
Campaigners urge green light for life-transforming treatment as one of first babies given wonder drug enjoys happy and healthy Christmas at home
Regulators will decide within weeks whether a new treatment for patients with a rare neuromuscular disease will be available in Scotland.
The treatment – risdiplam, which can be taken at home as a syrup – will be considered by the Scottish Medicines Consortium on January 11 when the body will decide whether to recommend the drug is funded by the NHS for the treatment of spinal muscular atrophy (SMA).
Also called Evrysdi, it has been approved for use south of the border for those with SMA aged two months and older, as part of a deal known as a Managed Access Agreement (MAA) between NHS England and the drug’s manufacturer, Roche.
The list price of risdiplam is £7,900 per 80ml vial, but a discounted deal is reported to have been struck to make it available to the NHS. It is especially important for those with SMA who cannot receive either of the two existing treatments –
Spinraza (also known as nusinersen) and Zolgensma.
Liz Ryburn, SMA UK’S support team manager, said: “We look forward to continuing to take part in the Scottish Medicines Consortium’s considerations as to whether they will recommend that risdiplam is funded by the NHS for the treatment of SMA.
“We will join the committee on January 11 to continue to represent the patient voice. A positive decision about this home-based oral daily medication would open the door and offer life-changing potential to those who have not yet had the opportunity to access treatment.”
Ryburn also applauded progress so far with Zolgensma, which was used to treat seven-month-old Isabella Winfield. The Sunday Post told in June how she had become the second Scot to get the drug.
Ryburn said: “Isabella is doing fantastically and we are delighted. All the children treated so far – some 65 in England and two in Scotland – are responding and it is looking very positive. Six or seven years ago, these children would have been unlikely to survive beyond the age of two years.
“While the Zolgensma treatment is going very well, to maximise its potential benefit we are relying on early diagnosis, and sadly that does not always happen. We therefore urge the UK National Screening Committee to swiftly progress their review of the case for newborn screening in the UK.
“In 2018, they did not recommend screening for the condition because at that time the criteria for adding SMA to the panel of screening tests were not met. These criteria include the availability of treatments, cost effectiveness and acceptability to the community. Now that things have moved on for SMA with new treatments like nusinersen, Zolgensma, and risdiplam, we are hopeful of change.”