1.1 Risdiplam is recommended as an option for treating 5q spinal muscular atrophy (SMA) in people 2 months and older with a clinical diagnosis of SMA types 1, 2 or 3 or with pre-symptomatic SMA and 1 to 4 SMN2 copies. It is recommended only if the conditions of the managed access agreement are followed.
Why the committee made these recommendations
SMA is a rare genetic condition and there is an unmet need for effective treatments that can slow disease progression.
Clinical evidence shows that risdiplam improves motor function in SMA types 1 to 3. There is some evidence suggesting that people with type 1 SMA who have risdiplam live for longer. There is also some evidence suggesting risdiplam may be effective for people with pre-symptomatic SMA. But there is no direct evidence comparing risdiplam with usual care for type 1 SMA. And although it's likely that risdiplam has long-term benefits, there is no long-term evidence, so this is uncertain.
The cost-effectiveness estimates are higher than what NICE usually considers an acceptable use of NHS resources. So risdiplam cannot be recommended for routine use in the NHS. But because of the unmet need for effective treatments for SMA, risdiplam is recommended through a managed access agreement while more data is collected to address the uncertainties in the evidence.