3 July 2019 - More children with the rare genetic disorder spinal muscular atrophy can now be treated with Spinraza after NICE today published amended draft guidance following a proposal for extending the terms of the managed access agreement between NHS England and Biogen for funding it.

The company made the proposal taking into consideration further clinical evidence. The agreement has also been refined in light of a number of requests for clarification.

The most significant change to the agreement is that it will now include paediatric patients who have recently (in the previous 12 months) lost the ability to walk independently. This replaces the previous criteria that patients, who had previously gained ambulation, should still be able to walk independently at the start of their treatment with Spinraza.

Read NICE press release