Last week outside Parliament I met with my constituent Julie Gill and her son nine year old son Oscar, who was diagnosed at just seven with Freidrich’s Ataxia. They had come to London to join other families campaigning for the drug Omaveloxone, sold under the brand name Skyclarys, to be made available in the UK.
Freidrich’s Ataxia is a rare, life-limiting genetic disorder that impacts around 1200 people in the UK. It is an inherited progressive disorder that damages the spinal cord, peripheral nerves, cerebellum and heart. The average life expectancy for someone with the condition is just 37 years old but Omaveloxolone can slow down disease progression by between 50% and 55%, which is why it is so important families can access the drug. It is already available in 40 other countries around the world.
Despite being licensed by the MHRA for over-16s in April 2025, manufacturers Biogen withdrew its evidence submission from NICE, leaving families unable to get access to the drug here. This has left some families having to source supplies abroad, though the annual price of treatment is around £298,000 a year.
A petition on the government’s website into this recently gained 103,603 signatures, which meant it had to be considered for a parliamentary debate, something the Petition’s Committee will be doing shortly. I’ll be joining that debate if held and will also be raising this with Ministers and the manufacturers. Freidrich’s Ataxia sufferers like Oscar don’t have time for this to drag on without resolution.
The campaign was also featured on ITV News, which you can see below.