UK – NICE recommends Sprinraza for 5q spinal muscular atrophy

The National Institute for Health and Care Excellence (NICE) has recommended funding for Spinraza (nusinersen) on the National Health Service (NHS).

The recommendation is for infants, children and adults with 5q spinal muscular atrophy and will be available through a scheme known as a Managed Access Agreement (MAA), meaning that patients will be able to get Spinraza while more long-term data on its efficacy is gathered.

The evaluation based on the largest body of clinical evidence currently available across all types of SMA, with six years of data including more than 300 patients treated with pre- symptomatic, infantile and later-onset, or types I and II SMA.

Muscular Dystrophy UK and Spinal Muscular Atrophy UK are now calling on NICE to formally recommend the treatment and for Biogen and NHS England to urgently implement the MAA so patients can access the drug quickly, and for Wales and Northern Ireland, which generally follows NICE’s guidance, to follow suit.

The announcement is “fantastic news that gives families home”, said Catherine Woodhead, chief executive of Muscular Dystrophy UK…