First-in-class therapy approved for schizophrenia

The US Food and Drug Administration (FDA) has approved Cobenfy (xanomeline and trospium chloride) for schizophrenia in adults.

“Today’s landmark approval of our first-in-class treatment for schizophrenia marks an important milestone for the community, where after more than 30 years, there is now an entirely new pharmacological approach for schizophrenia — one that has the potential to change the treatment paradigm,” shared Dr Chris Boerner, PhD, Board Chair and Chief Executive Officer at Bristol Myers Squibb.

“We will be running the first UK trial of this compound in Oxford, starting in 2025,” shared Dr Robert McCutcheon, Wellcome Clinical Research Career Development Fellow, Department of Psychiatry, University of Oxford.

The oral muscarinic agonist is the first authorised antipsychotic schizophrenia drug that targets cholinergic receptors instead of dopamine receptors, which has been standard of care for this indication, according to the FDA.

Bristol Myers Squibb highlighted that Xanomeline is a dual M ₁ – and M ₄ -preferring muscarinic receptor agonist. Trospium chloride is a muscarinic receptor antagonist that primarily has action in the peripheral tissues to the blood-brain barrier.

Clinical data behind the approval of Cobenfy

The agency’s decision is based on findings from the EMERGENT clinical programme. Results from the Phase III EMERGENT-2 and EMERGENT-3 trials showed that Cobenfy enabled statistically significant reduction in the symptoms of schizophrenia in participants.

“This approval offers a new alternative to the antipsychotic medications people with schizophrenia have previously been prescribed,” commented Dr Tiffany Farchione, Director of the Division of Psychiatry, Office of Neuroscience in the FDA’s Center for Drug Evaluation and Research.

With its differentiated mechanism of action, “approval of Cobenfy is a transformative moment in the treatment of schizophrenia because, historically, medicines approved to treat schizophrenia have relied on the same primary pathways in the brain. By leveraging a novel pathway, Cobenfy offers a new option to manage this challenging condition,” explained Dr Rishi Kakar, Chief Scientific Officer and Medical Director at Segal Trials and investigator in the EMERGENT programme.