More children with the rare genetic disorder spinal muscular atrophy (SMA) can now be treated with Spinraza after NICE published on July 3, 2019, amended draft guidance following a proposal for extending the terms of the managed access agreement between NHS England and Biogen for funding it.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 managed access agreement has also been refined in light of a number of requests for clarification.
The most significant change to the managed access agreement is that it will now include pediatric patients who have recently (in the previous 12 months) lost the ability to walk independently. This replaces the prior criteria that patients, who had previously gained ambulation, should still be able to walk independently at the start of their treatment with Spinraza.
It is estimated there are between 600 and 1200 children and adults in the UK living with SMA.
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