New NHS treatment available for first time for patients with a rare neuromuscular disorder

Patients at Great Ormond Street Hospital (GOSH) and across the country are to benefit from a new drug on the NHS that targets the underlying cause of spinal muscular atrophy (SMA).

The approval of the drug, called Nusinersen or Spinraza, follows successful trials at GOSH and other centres around the world that have shown the drug can slow the effects of SMA in some cases, allowing babies and toddlers to develop stronger muscles and survive for longer without breathing support.

NHS England have negotiated a deal with the drug’s manufacturer Biogen to be able to make the drug widely available.

Professor Francesco Muntoni, a Paediatric Neurologist at Great Ormond Street Hospital, said:

“We are delighted with this long-awaited decision. Great Ormond Street Hospital played a significant role in a highly successful multi-centre global trial which saw striking results where the drug delivered visible improvements in motor and respiratory outcomes of our patients.

“For many children with SMA, this treatment could be the silver bullet, offering children and families affected by this devastating condition, a therapy that could increase their survival when there has otherwise been no other option.”

SMA affects the nerves in the spinal cord, making muscles weaker and causing problems with movement, breathing and swallowing. Where it develops in babies and toddlers, it can significantly reduce life expectancy.

The new funding agreement now paves the way for the treatment to be made available to the youngest and most severely affected (SMA type 1) patients immediately by Biogen. For older babies, children and young adults with less severe symptoms (SMA types 2 and 3), the NHS will begin to provide Nusinursen shortly after NICE’s guidance is published, once the services to deliver them are established.