Biogen Inc. (Nasdaq: BIIB) announced that the European Commission (EC) has granted marketing authorization in exceptional circumstances for tofersen for the treatment of adults with amyotrophic lateral sclerosis (ALS) associated with a mutation in the superoxide dismutase 1(SOD1-ALS) gene, while maintaining orphan drug status for the therapy. Tofersen is the first therapy approved in the European Union to target the genetic cause of ALS, which is also known as motor neuron disease (MND).
"The European Commission's approval of tofersen is a testament to the unwavering dedication of the ALS community - people living with the disease and their loved ones, researchers, clinicians and advocates - who have worked together over the past two decades to bring this important new therapy to the community of ALS patients with mutations in the SOD1gene," - said Dr. Stephanie Fradette, head of Neuromuscular Development at Biogen. "We are working with the medical community and local authorities to make the treatment available as soon as possible to people in the region living with amyotrophic lateral sclerosis (ALS) with a mutation in the SOD1 gene."
The marketing authorization for tofersen has been granted under an approval procedure in exceptional circumstances. The use of such a procedure is recommended when the benefit-risk assessment of a treatment is determined to be favorable, but due to the rarity of the disease it is unlikely that comprehensive data can be obtained under normal conditions of use. The European Medicines Agency (EMA) has recommended maintaining orphan drug status for tofersen for the treatment of amyotrophic lateral sclerosis (ALS).
"The approval of tofersen represents a paradigm shift in the treatment of ALS with a mutation in the SOD1 gene, giving hope to patients and their loved ones who have long been waiting for a breakthrough," said Dr. Philip Van Damme, professor of neurology and director of the Reference Center for Neuromuscular Diseases at Leuven University Hospital in Belgium. "The European Academy of Neurology has confirmed new guidelines for the treatment of ALS, which say that tofersen should be used as a first-line drug in ALS patients with mutations in the SOD1gene."
The approval of tofersen is based on the totality of the evidence, including the targeted mechanism of action, biomarkers and clinical data. In the randomiz...
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