NHS deal on spinal muscular atrophy at home treatment

A life-changing drug that can improve mobility in children and adults with a rare genetic condition will be available on the National Health Service (NHS) following a new commercial deal, chief executive Amanda Pritchard announced.

Risdiplam, which is marketed by Swiss pharma giant Roche (ROG: SIX) under the trade name Evrysdi, will be used to treat hundreds of patients a year with spinal muscular atrophy (SMA), a rare and often fatal genetic disease that causes paralysis, muscle weakness and progressive loss of movement.

The drug has a list price of nearly £8,000 ($10,720) per dose, but following a deal struck between the manufacturer Roche and NHS England, the health service will have access to the drug at a price that is fair for taxpayers, the agency noted.