The European Medicines Agency (EMA) has recommended refusing marketing authorisation for Blarcamesine Anavex, a proposed treatment for Alzheimer’s disease, following a negative opinion adopted on 11 December 2025. The applicant company, Anavex Germany GmbH, can request a re-examination within 15 days of receiving the opinion.
Blarcamesine Anavex contains blarcamesine (as blarcamesine hydrochloride) and was intended to be supplied as hard capsules, to be used in addition to other treatments. During the evaluation, the company proposed narrowing the target population to adults with early Alzheimer’s disease, either mild cognitive impairment due to Alzheimer’s disease or early-stage mild dementia due to Alzheimer’s disease, specifically in people without a SIGMAR1 gene mutation.
The proposed mechanism of action was activation of the sigma-1 receptor protein, with the aim of supporting nerve cell function and reducing damage linked to inflammation, in turn slowing cognitive decline.
The application relied mainly on results from a 48-week study involving 462 adults aged 60 to 85 years with early Alzheimer’s disease, comparing blarcamesine with placebo. The co-primary outcomes assessed change in cognition using ADAS-Cog13 and change in ability to perform daily activities using ADCS-ADL, alongside subgroup analyses including participants without SIGMAR1 mutations.
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EMA concluded that the main study did not demonstrate effectiveness and safety in the proposed SIGMAR1 non-mutation subgroup. The trial did not meet its main objective of showing improvement across both primary measures, and EMA highlighted methodological issues that raised concerns about the validity of the results, preventing a positive conclusion on efficacy. On safety, EMA cited limitations in the safety database and how safety data were collected, and noted a high proportion of treatment discontinuations, mainly due to central nervous system side effects, raising concerns about tolerability. EMA also identified quality concerns, stating that available information did not rule out the formation of nitrosamine impurities. Although the company applied for conditional marketing authorisation during the procedure, EMA said the criteria were not met and recommended refusal.
This news item has been summarised using AI and checked by humans before publication.
Sources
ema.europa.eu
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