Last updated 2nd June 2017.
European Commission Approval
On 1st June, the European Commission approved marketing authorisation of Spinraza "for the treatment of 5q spinal muscular atrophy", which includes SMA Types 1, 2 and 3. Read more here.
In the UK, the availability of the drug to anyone other than children eligible via the Expanded Access Programme (EAP) for SMA Type 1 now depends on a recommendation by the National Institute for Health and Care Excellence (NICE), NHS England, the Scottish Medicines Consortium and other authorities in the devolved nations to decide whether to fund the drug in England, Scotland, Wales and Northern Ireland. Availability may also depend on the treatment readiness of specialist centres.
Biogen is waiting to hear from NICE if this is to happen and on the appraisal route of nusinersen (see below about the recommended HST route). This should be known in July at which point NICE will formally invite Biogen to make a submission.
You can read more about the NICE process and what has happened thus far here as well as what happens for the rest of the UK.
Drug Funding and Approvals Process - changes to HST criteria
In the NICE draft scoping workshop in January 2017, patient representatives and clinicians put forward the opinion that nusinersen should be assessed as a Highly Specialised Technology (HST).
We are concerned that NICE and NHS England have jointly announced that they are pressing ahead with changes to the drug funding and approvals process in England which threaten the possibility of nusinersen getting to the point of being funded by the NHS. You can read: Genetic Alliance’s summary of the changes and what their impact would be here; a report in the Pharma Times here; a report from the All Party Parliamentary Group for Muscular Dystrophy that we attended on Wednesday 29 March here and our view on what this could mean here.