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Under threat from Novartis, Biogen hails new data from SMA drug Spinraza

July 02, 2019 / Richard Staines

Under threat from a gene therapy from Novartis, Biogen is making the case for its drug Spinraza for the muscle wasting disease spinal muscular atrophy, with new data that it hopes persuades doctors to prescribe it. Despite its steep list price of around £450,000 for the first year of treatment NHS England and NICE have struck a deal with Biogen that will allow patients access under a managed access agreement. This involves a confidential price cut and funds treatment for a time-limited period until further data is available on Spinraza’s effectiveness. The latest data from the NURTURE study could well help NICE with its decisions about how to fund Spinraza in the longer term. Results in NURTURE involve data from the longest study SMA in pre-symptomatic infants, involving up to 45.1 months of analysis. Data demonstrate efficacy and safety in patients treated pre-symptomatically in comparison with the natural history of the disease. The 25 patients treated with Spinraza had c...