Biogen unveils promising new data for Spinraza in pre-symptomatic infants with spinal muscular atrophy

pharmafile | July 2, 2019 | News story | Manufacturing and Production, Research and Development Biogen, Spinraza, pharma, spinal muscular atrophy 

Biogen has introduced new data on the efficacy of Spinraza in the treatment of pre-symptomatic infants in spinal muscular atrophy (SMA), bolstering what the knowledge base emerging from the NURTURE study, the longest study of its kind in this condition and patient population.

The new data from the ongoing Phase 2 trial compared the efficacy of Spinraza in 25 patients who were most likely to develop SMA type 1 or 2 who had received their first dose of Spinraza before they were six weeks old, as opposed to those experiencing the natural history of the condition.

After up to 45.1 months of treatment, it was found that all patients were still alive without need for permanent ventilation, and 88% were able to walk independently with many doing so within the normal timeframe experienced by toddlers without the condition – something which SMA type 1 or 2 patients are never able to do in the natural history of the disease. In addition, the drug “demonstrated longer term efficacy up to nearly four years”, and patients displayed no loss of motor function while they progressed.

“These study results demonstrate the durable impact of pre-symptomatic, proactive treatment on transforming the natural course of this disease. We are seeing an extensive number of patients continually meeting child motor development milestones and making unprecedented gains in a previously hopeless and often fatal condition,” explained Dr Darryl De Vivo, Sidney Carter Professor of Neurology and Pediatrics at the Columbia University Irving Medical Center in New York. “Spinraza is setting patients on a path toward survival, greater mobility and independence, which is helping improve outcomes for patients of all ages.”

Kenneth Hobby, President of Cure SMA, also commented: “A few years ago, SMA patients had no treatment options and faced significant care challenges. However, the future of SMA has changed and especially with early treatment patients now have a very strong chance to reach age appropriate developmental milestones. These new data demonstrate the dramatic impact where children are now walking independently at four years of age, when the usual lifespan would be under two if untreated. This study provides additional evidence on the maintenance of these improvements. It’s critical that research in SMA continues to support the generation of real-world evidence in patients of all ages so that we better understand the long-term implications of SMA and treatment across all types.”

Matt Fellows

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