The draft guidelines reject NHS funding for the biogene Spinraza

The National Institute for Health and Care Excellence currently does not recommend that the NHS use Biogen's Spinraza to treat the rare hereditary spinal muscular dystrophy (SMA).
In its draft guidelines, the institute said the evidence showed "material benefits" for the drug, but the evaluation committee considered that there were "significant uncertainties, particularly around its long-term interests," which, together with Spinraza's "very high" costs, meant it could not be recommended as a cost-effective treatment option.
, however, NICE welcomed "further discussions" and noted that it was aware of Bigogen's intention to develop a proposal to manage access plans that could address uncertainty in the evidence and reduce the financial risk to the NHS.
in the UK, an estimated 1,200 to 2,500 children and adults live with the SMA, which leads to devastating muscular atrophy, which causes patients to lose the ability to move, breathe and swallow. People with the most severe symptoms usually die before the age of 2 and there is currently no active treatment for their underlying causes.
Spinraza is an anisotym oligonucleotide (ASO) used to treat SMA caused by mutations or deficiencies in the SMN1 gene located in chromosome 5q that causes the lack of SMN proteins. is the first to address the cause of motor neurone degeneration in patients with the disease.
the therapy was accelerated in Europe in May 2017 after an accelerated evaluation, based on data from two key multi-center controlled studies that show clinically significant efficacy and beneficial benefit-risk characteristics, according to the company.
earlier this year, Biogen said it was "optimistic" that patients with rare spinal diseases would be able to access Spinraza quickly and widely through the NHS. Although England and Wales now appear more uncertain, the Scottish Medicines Union has accepted the drug for use on the NHS in Scotland. (China Medicine 123 Network)