US Alzheimer’s drug pioneer Anavex presses-on, despite regulatory setback

Alzheimer’s drug pioneer Anavex is pressing ahead with plans to secure approval for its ground-breaking treatment despite a recent setback.

Earlier this year it lodged an appeal against the European Medicines Agency (EMA) decision to reject its application for drug approval in December 2025.

But, Anavex has now withdrawn this appeal as it seeks to address the points raised by the EMA’s Committee for Medicinal Products for Human Use (CHMP).

The Nasdaq-listed company says it is engaged in further discussions with European and US regulators as it looks to secure approval for its daily Alzheimer pill Blarcamesine.

In a press release it said: “Anavex now has to take note of the differing assessment of the CHMP at the EMA and will consider the constructive feedback received from the CHMP and focus on gathering additional data and conducting further analyses to address the points raised by the CHMP.”

Dr Christopher Missling, president and chief executive officer of Anavex, said: “We remain committed about advancing the development of innovative oral therapies for patients living with both neurodegenerative diseases, including early Alzheimer’s and Parkinson’s disease and neurodevelopmental disorders as we continue our engagement with regulatory authorities,”

This comes as it continues to work with US regulators the Food and Drug Administration to secure approval in its domestic market.

Dr Missling told Agetech World: “Alzheimer’s disease remains one of the most urgent and challenging public health issues of our time, and addressing it requires close collaboration across the healthcare ecosystem.

“We value our ongoing dialogue with the FDA as we work to advance our program responsibly and transparently. Their guidance is essential as we pursue innovative approaches aimed at meeting the significant unmet needs of patients, families, and caregivers affected by this devastating condition.”

In rejecting Blarcamesine the EMA said it had ‘failed to demonstrate effectiveness and safety’.

In response to Agetech World on recent developments, the EMA said: “Anavex Germany withdrew its application for a marketing authorisation of Blarcamesine Anavex for the treatment of Alzheimer’s disease and dementia (cognitive impairment).

“The company withdrew the application on March 25, 2026. The European Medicines Agency had recommended refusing the marketing authorisation in December 2025.

“The company had then requested a re-examination of the Agency’s opinion, but it withdrew the application before the re-examination had finished.”

In a In a Phase IIb/III trial of Blarcamesine over one-third of patients with mild Alzheimer’s – those with a Mini-Mental State Examination (MMSE) score of between 20 and 28 – experienced a slowing of their ‘cognitive decline’.

And, for the majority of the population – those with the common sigma-1 gene – the results were even more dramatic, slowing decline by 49.8%