Spinraza is the first and only treatment for patients with the rare inherited muscle-wasting condition spinal muscular atrophy (SMA). Without access to the drug, life expectancy for those with SMA Type 11 is rarely longer than two years. The drug is also holding promise for people with other forms of SMA.
At a special meeting on 3 April, families affected by the condition will make a final plea to the Scottish Medicines Consortium (SMC) to approve Spinraza for use on the NHS in Scotland2.
Muscular Dystrophy UK is warning that unless the SMC approves Spinraza, families who could benefit from a proven treatment will be made to suffer needlessly.
Robert Meadowcroft, Chief Executive at Muscular Dystrophy UK, said:
“SMA can be devastating, and Spinraza offers families a rare glimmer of hope. Its most severe form means parents have to see their child gradually lose their ability to crawl, move, breathe and swallow while there is a treatment out there which could help. Nothing can prepare you for the emotional turmoil this causes.
“Spinraza is not a cure, but it can buy families time to spend with their loved ones. Some children who have received Spinraza have seen their muscle strength improve and have already lived long enough to crawl, and even walk. For parents, this is priceless.
“The SMC has a chance to hand patients the lifeline they so desperately need. If they squander this opportunity to bring hope into the lives of people affected by muscle-wasting conditions, families will be made to pay a very heavy price.”
Spinraza proved so effective in a clinical trial for children with SMA Type 1 that the trial was stopped early so that all children affected could access the treatment. Treatment is now being delivered to children in Scotland via a special compassionate access scheme, with the drug being provided free by pharmaceutical manufacturer Biogen and the NHS in Scotland funding the costs of administering the treatment. However, it is a temporary scheme, and only supports a small number of those with SMA who could benefit.
Hayleigh Barclay, 30, from Prestwick, Ayrshire, who has SMA Type 2, said:
“It’s hard to overstate how much of a lifeline Spinraza would be for many people. I’m a very determined, ambitious person with many goals, but having to constantly battle against the effects of SMA severely impacts on your quality of life.
“I enjoy going to rock concerts, socialising with friends and spending time with my family. I’m also studying for a PhD, but all of these things are becoming increasingly difficult. I want to achieve so much in my life, and getting access to Spinraza would help to maintain my health in order for me to achieve my goals.
“Knowing there is a treatment out there with the potential to make a real difference is life changing and gives hope to many people. The SMC has the power to transform the future of the families whose lives have been affected by SMA.”
Find out more on Muscular Dystrophy UK’s Fast Track campaign to speed up access to treatment at: www.musculardystrophyuk.org/fast-track.