The US Food and Drug Administration opted for accelerated approval, rather than regular approval, for Biogen, Inc. and Ionis Pharmaceuticals, Inc.’s Qalsody (tofersen) in a genetically defined subset of amyotrophic lateral sclerosis patients, banking that clinical benefit will be confirmed in an ongoing trial of presymptomatic individuals.
Biogen/Ionis’ Tofersen Wins Accelerated Approval In ALS Subgroup – A Significant But Narrow Precedent
US FDA designates ongoing ATLAS trial in presymptomatic individuals with SOD1 mutation as confirmatory study. ‘Classic Biogen’ approval is first in ALS under the expedited pathway and could lead to more drugs reaching market on basis of a reduction in plasma neurofilament light chain concentration.
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The Pink Sheet's list of EU centralized approvals of new active substances has been updated to include four new products, one of which is Vimkunya, Bavarian Nordic's chikungunya vaccine.
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Levodopa/carbidopa (ND0612), Mitsubishi Tanabe Pharma’s investigational drug-device combination therapy for the treatment of motor fluctuations in people with Parkinson’s disease, is among the latest products that have been filed for review by the European Medicines Agency for potential EU marketing approval.
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Japan recommends eight new drugs for approval, including two for cardiomyopathy, and will also raise prices for multiple products, including Enhertu, on 1 April.
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Unofficial January results showed the US FDA continued to issue full and tentative ANDA approvals in line with previous months, but recent layoffs may not help the agency maintain that pace.
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Novartis is planning to file EU and US marketing applications for an intrathecal formulation of its spinal muscular atrophy gene therapy, Zolgensma, in H1 2025.
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Sponsors of three drugs that are in the final stages of the EU regulatory review cycle are due to make the case for marketing approval before the European Medicines Agency.
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The European Medicines Agency is this week set to issue an opinion on whether pan-EU marketing should be granted to Lilly’s Alzheimer’s disease drug donanemab.