BIOGEN ANNOUNCES NEW DATA FURTHER ESTABLISHING SPINRAZA? (NUSINERSEN) AS A FOUNDATION OF CARE IN SPINAL MUSCULAR ATROPHY FOR A BROAD RANGE OF PATIENTS
[caption id="attachment_9277" align="aligncenter" width="747"] Press Release[/caption]
July 1, 2019 at 7:30 AM EDT
- New results from the NURTURE study demonstrate that pre-symptomatic infants treated with SPINRAZA are achieving motor milestones that are unprecedented in the natural history of the disease, including 100 percent of children sitting without support and 88 percent walking independently
- Results add to the body of evidence supporting the safety and long-term efficacy of SPINRAZA and demonstrate that treatment results in continuous improvement in attaining motor milestones
- SPINRAZA is the only treatment approved for infants, children and adults with evidence across a broad range of patients and real-world experience treating more than 7,500 patients
- 100 percent were alive without a need for permanent ventilation.
- The median age of the study participants was nearly three years old. The majority of untreated patients with SMA Type 1 never reach their second birthday without permanent ventilation.
- 100 percent of the infants were sitting independently, in comparison to the natural history of this disease where no patients with SMA Type 1 would be able to do so and patients with SMA Type 2 would need assistance.
- 88 percent of the infants were walking independently with many of them doing so in the normal timeframe for a toddler. In the natural history of SMA, patients with SMA Type 1 or Type 2 are never able to walk independently.
- Patients were approaching the maximum mean score of 64 on the CHOP INTEND measure of motor function? 63.4 for patients with 3 SMN 2 copies (n=10) and 62.1 for those with 2 SMN 2 copies (n=15), demonstrating the powerful impact of early SPINRAZA treatment.
- SPINRAZA demonstrated longer term efficacy up to nearly 4 years, with participants continuing to make progress and showing no signs of loss of motor function.
- SPINRAZA was well-tolerated with no new safety concerns identified after up to nearly 4 years of treatment.
- Finkel, Richard S., et al. ?Nusinersen versus Sham Control in Infantile-Onset Spinal Muscular Atrophy.??New England Journal of Medicine, vol. 377, no. 18, 2017, pp. 1723?1732., doi:10.1056/nejmoa1702752.
- Lefebvre S, Burglen L, Reboullet S, et al. Identification and characterization of a spinal muscular atrophy-determining gene.?Cell.1995;80(1):155-165.
- Mercuri, Eugenio, et al. ?Nusinersen versus Sham Control in Later-Onset Spinal Muscular Atrophy.??New England Journal of Medicine, vol. 378, no. 7, 2018, pp. 625?635., doi:10.1056/nejmoa1710504.
- Basil T.?Darras,?et al. ?Nusinersen in later-onset spinal muscular atrophy.? Neurology,?May 2019,?92?(21)?e2492-e2506;?DOI:10.1212/WNL.0000000000007527
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