Photo Courtesy: twitter/ Spinal Muscular Atrophy UK

The National Institute for Health and Care Excellence (NICE) today recommended Nusinersen (Spinraza) made by Biogen for NHS funding.

It is the first ever treatment for children with the rare genetic disorder, spinal muscular atrophy (SMA). The recommendation is following a successful negotiation between NHS England and Biogen to make this treatment available for those whose clinician think they would benefit.

“The committee has recognised that Nusinersen is a promising treatment that has been shown to improve a range of outcomes important to patients. But it also recognised that there are significant uncertainties, particularly around its long-term benefits,” said Meindert Boysen, director of the Centre for Health Technology Evaluation at NICE.

SMA affects the nerves in the spinal cord causing weaker muscles, progressive loss of movement and, difficulty in breathing and swallowing. Between 600 and 1,200 children and adults are currently living with this condition in the UK.

“The NHS has now reached one of the most comprehensive deals in the world, which allows us to assess real-world evidence of its long term benefits,” Simon Stevens, NHS England chief executive, said.