- Label update includes data from four clinical studies including long-term safety and efficacy data across a broad Spinal Muscular Atrophy (SMA) population
- Data from over 300 patients across a broad range of ages and types of Spinal Muscular Atrophy (SMA) who have received treatment for up to 5 years have been added to the label.
BAAR, Switzerland, June 29, 2019 (GLOBE NEWSWIRE) -- Biogen (BIIB) today announced an update to the European label for nusinersen, incorporating longer-term data from four studies (NURTURE, EMBRACE, SHINE and CS3A). These studies, which looked at pre-symptomatic, infantile-onset and later-onset treatment, further confirm the benefits of early treatment and durability of treatment effect of nusinersen. No new safety concerns were identified.
Collectively, these data demonstrate that treatment with nusinersen results in continuous improvement or stabilization of disease activity for patients of all ages. Whilst the benefits are greatest with earlier treatment initiation, the SHINE data, which followed patients from the pivotal CHERISH and ENDEAR studies, also demonstrates that patients starting treatment at a later stage can still gain significant improvements in motor function and abilities that can help them in their day-to-day lives. At the time of interim analysis of SHINE, length of treatment with nusinersen was up to 474 days for patients enrolled from CHERISH and up to 592 days for those from ENDEAR.
“These data confirm the longer-term safety and efficacy profile of nusinersen and add to the impressive and growing body of clinical evidence including more than 300 patients in a broad range of ages and types of SMA,” said Ivana Rubino, Executive Medical Director and Global Head of Neuromuscular Medical Affairs at Biogen. “Over 7,500 infants, teens and adults have now been treated with nusinersen worldwide. Biogen is also continuing to help build several worldwide disease registries and real world data initiatives which will continue to deliver important long-term insights into SMA and its treatment.”
The European label update also includes data from EMBRACE which examined both infantile and later-onset SMA, final results from the CS3A study (which investigated the use of nusinersen in symptomatic patients with infantile-onset SMA) as well as data from the NURTURE study. NURTURE is an ongoing, open-label study of pre-symptomatic infants genetically diagnosed with SMA, the latest results of which are being presented at the CureSMA and EAN Congresses in the coming days.
For the full European label update please visit: https://www.ema.europa.eu/en/documents/product-information/spinraza-epar-product-information_en.pdf
About SPINRAZA® (nusinersen)1-4
Nusinersen is the first therapy approved to treat infants, children and adults with spinal muscular atrophy (SMA) and is available in more than 40 countries. As of March 31, 2019, more than 7,500 individuals have been treated with nusinersen for up to six years, based on patients across the post-marketing setting, Expanded Access Program (EAP) and clinical trial participants. Nusinersen is the only SMA treatment to combine unsurpassed real-world experience and the highest level of clinical evidence across a broad spectrum of patient populations.
SMA is a rare, genetic, neuromuscular disease that is characterized by a loss of motor neurons in the spinal cord and lower brain stem, resulting in severe, progressive muscle atrophy and weakness. Approximately one in 10,000 live births have a diagnosis of SMA and people of all ages are impacted by the disease. It is a leading genetic cause of infant mortality.
Nusinersen, a foundation of care in SMA, is an antisense oligonucleotide (ASO) designed to target a root cause of SMA by increasing the amount of full-length SMN protein, which is critical to maintaining motor neurons. It is administered by intrathecal injection into the fluid surrounding the spinal cord where motor neurons reside to deliver the treatment where the disease starts.
Nusinersen currently maintains the largest clinical data set in SMA based on data from over 300 patients across a broad range of SMA populations demonstrating a favorable benefit: risk profile. Nusinersen was evaluated in two randomized, double-blind, sham-controlled studies (ENDEAR and CHERISH) in infantile and later-onset SMA patients and supported by open label studies in pre-symptomatic infants (NURTURE) and individuals who were treated into adulthood with later-onset SMA (CS2/12). The most common adverse events observed were headache, vomiting, and back pain. Serious infections of hydrocephalus and meningitis have been observed in the post-marketing setting. Renal toxicity and coagulation abnormalities, including acute severe low platelet counts, have been observed after administration of some ASOs. Laboratory tests can monitor for these signs.
Biogen licensed the global rights to develop, manufacture and commercialize nusinersen from Ionis Pharmaceuticals, Inc. (IONS), a leader in antisense therapeutics. Biogen and Ionis conducted an innovative clinical development program that moved nusinersen from its first dose in humans in 2011 to its first regulatory approval in five years.
At Biogen, our mission is clear: we are pioneers in neuroscience. Biogen discovers, develops, and delivers worldwide innovative therapies for people living with serious neurological and neurodegenerative diseases as well as related therapeutic adjacencies. One of the world’s first global biotechnology companies, Biogen was founded in 1978 by Charles Weissmann, Heinz Schaller, Kenneth Murray, and Nobel Prize winners Walter Gilbert and Phillip Sharp, and today has the leading portfolio of medicines to treat multiple sclerosis, has introduced the first approved treatment for spinal muscular atrophy, and is focused on advancing neuroscience research programs in MS and neuroimmunology, Alzheimer’s disease and dementia, movement disorders, neuromuscular disorders, acute neurology, neurocognitive disorders, pain, and ophthalmology. Biogen also commercializes biosimilars of advanced biologics.
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- 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