SMA Typ 1-3: Safety and Treatment Effects of Nusinersen in adults and infants

CAMBRIDGE, Mass., April 12, 2019 (GLOBE NEWSWIRE)Biogen (Nasdaq: BIIB) today announced it will present new data illustrating the rapidly progressive nature of spinal muscular atrophy (SMA) in adults, adolescents and older children at the Muscular Dystrophy Association (MDA) Clinical and Scientific Conference in Orlando, Florida (April 13-17, 2019). Other Biogen presentations will highlight the benefits of pre-symptomatic treatment with data from the NURTURE study, part of the SPINRAZA® (nusinersen) global clinical trial program, and findings on the role of neurofilament as a potential biomarker for predicting motor function in SMA.

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  1. “The data show that, if left untreated, people with all types of SMA continue to experience increased health challenges, and that slowing the progression of the disease, even for those with less severe forms, is critical to their ongoing health,” said Wildon Farwell, M.D., senior medical director, clinical development at Biogen. “With more than 6,600 individuals treated with SPINRAZA across the post-marketing setting, expanded access program and clinical trials and over six years of clinical data, we continue to expand our in-depth knowledge of the disease and to gain real-world experience on long-term safety and efficacy. We plan to continue advancing research to help shape care for individuals with SMA today and in the future.”

    Read more about the new presentations on SMA and SPINRAZA

    #Behandlung mit Spinraza bei Erwachsenen

  2. New Data by Biogen Treatment with SPINRAZA resulted in clinically meaningful improvements in motor-function and survival in individuals with spinal muscular atrophy SMA Type 1, 2 or 3

    Data from CHERISH-SHINE, which evaluated individuals with later-onset SMA (n=125, most likely to develop SMA Type 2 or 3), demonstrated that earlier treatment resulted in greater improvements in motor function and continued improvement or stabilization of motor function scores. Mean change in Hammersmith Functional Motor Scale Expanded (HFSME) score was 3.7 for participants treated with SPINRAZA in SHINE and CHERISH. This compared to a positive 0.4 change from baseline among participants in the CHERISH sham-control arm and who received SPINRAZA in SHINE. During CHERISH, sham-control participants experienced a 0.4 decline in HFSME. Four participants in the youngest age group in the study (n=39, less than 3.69 years) were walking independently at Day 690 compared to no patients at baseline. There were no new safety findings. Patients were followed for up to four years, adding to the long-term safety profile of SPINRAZA.

    Data from the NURTURE study showed that pre-symptomatic treatment of infantile-onset SMA (n=25, most likely to develop SMA Type 1 or 2) resulted in motor milestone achievements more consistent with normal motor development. Evaluations were conducted at a median age of 26 months. All infants were able to sit without support and 88 percent could walk with assistance and 77 percent could walk without assistance. All infants were alive and none required permanent ventilation. No new safety concerns were identified. In the natural history of SMA, individuals are unable to sit without support (SMA Type 1) or walk (SMA Type 1 or 2) and often require respiratory and nutritional interventions in order to live past the age of two (SMA Type 1). The May 2018 data was previously presented.

  3. Biogen Inc. announced that the journal Neuromuscular Disorders has published data from NURTURE, the first study investigating a treatment targeting the underlying cause of spinal muscular atrophy (SMA) in infants treated pre-symptomatically. Data from the NURTURE study demonstrated that infants who initiated treatment with SPINRAZA prior to the onset of clinical symptoms attained unparalleled results compared to the natural history of the disease.

    NURTURE is an ongoing, Phase 2, open-label study of 25 infants with the genetic diagnosis of SMA (most likely to develop SMA Type 1 or 2) who received their first dose of SPINRAZA in the pre-symptomatic stage and before six weeks old. The study, conducted at 15 sites in seven countries, has results up to 45.4 months. When compared with the natural history of the disease, the results are dramatic in their impact on changing the course of SMA.

    Study results underscore the established safety profile, proven efficacy and durability for up to nearly four years of SPINRAZA

    Read more

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