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Home Industry News Biogen highlights benefits of new spinal muscular atrophy therapy

Biogen highlights benefits of new spinal muscular atrophy therapy

14th October 2016

Biogen has announced results from several clinical trials showing the potential benefit of its investigational treatment nusinersen against spinal muscular atrophy.

Presented by Biogen and Ionis Pharmaceuticals at the 2016 World Muscle Society Congress, initial safety data from the phase III ENDEAR study showed a favourable safety profile with no treatment-related adverse events among patients with infantile-onset spinal muscular atrophy.

Further results from the ongoing phase II open-label study NURTURE also demonstrated the drug's beneficial effect in infants prior to the onset of symptoms.

Wildon Farwell, senior director for spinal muscular atrophy clinical development at Biogen, said: "We continue to be encouraged by the consistently positive results with nusinersen across our clinical programme, including our first data in infants treated before they show signs of the disease."

Biogen has completed the rolling submission of a new drug application for the approval of nusinersen to the US Food and Drug Administration with a marketing authorisation application to be filed with the European Medicines Agency in the coming days.

Potentially, nusinersen could become the world's first approved treatment for spinal muscular atrophy.

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