The Scottish Medicines Consortium are the body who authorise the use of new drugs in NHS Scotland. They have been evaluating a new drug used to treat Spinal Muscular Atrophy, an inherited condition which attacks the motor neurones in the spinal cord, causing muscle wasting. The new drug, called Spinraza, was approved for limited use for infants who have this condition, but why should this be of interest to the HD community?

Spinraza is an antisense oligonucleic acid (ASO) and was developed by IONIS, one of the pharmaceutical companies currently developing a similar type of drug for HD and it’s good news because, like the treatment for HD, this drug is expensive and one of the hurdles it will need to clear, if it is shown to be successful in its phase three clinical trials, early 2019, is approval by the SMC. They will undertake a cost/benefit analysis to decide if NHS Scotland should fund the treatment. We’ve closely followed this drug because, while it is treating a very different condition, the shared underlying technology and cost, is of interest and a decision not to fund Spinraza might have raised some new questions.

What we should take away from this, is that it’s not enough for the IONIS HD gene silencing treatment to do well in its phase three clinical trials, we also need to build a strong economic case too.