NICE, the health technology assessment (HTA) body for England and Wales, has started to review data that was collected through a managed access agreement for Biogen’s Spinraza (nusinersen) for treating the rare genetic disorder of spinal muscular atrophy. The review could see the patient population eligible to receive the drug expanded to include SMA type III patients who cannot walk.
The review is restricted to SMA type III patients who are unable to walk; this group of patients was excluded from the NICE guidance on the use under the National Health Service of Spinraza that was published in May 2019. Also see "UK Rare Disease Drug Review Reform Urged As Spinraza Finally Gets NICE OK" - Pink Sheet, 16 May, 2019
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