NHS England will offer a promising novel life-prolonging treatment for children with a rare genetic condition called spinal muscular atrophy (SMA). The drug nusinersen (Spinraza) manufactured by Biogen is the first therapy developed to target the underlying cause of SMA.
An estimated 600 to 1200 children and adults in England and Wales live with SMA. While there is no definitive cure for the condition, evidence has shown nusinersen to slow the progression of SMA in certain cases, allowing the development of stronger muscles and longer survival without respiratory support.
NICE had previously not granted approval for routine use of nusinersen owing to uncertainties in its long-term efficacy and high cost. However, under the 'Managed Access Agreement' between NHS England and Biogen, NHS will fund nusinersen for a time-limited period, until further data on effectiveness are available.
Initially, the youngest patients and those with the most severe form of the condition (SMA type 1) will receive treatment. Older babies, children and young adults with symptoms of lesser severity (SMA types 2 and 3) will receive treatment upon publication of the final NICE guidance.
Meindert Boysen, Director of the Centre for Health Technology Evaluation at NICE, said: "Today’s announcement shows that, where companies show appropriate flexibility, it is possible to find a way to provide important treatments to patients in a way that is cost effective for the NHS and taxpayers."