FDA Approves New Kind of Drug for Schizophrenia

The first new type of medication in decades to help fight against schizophrenia was approved on Thursday by the U.S. Food and Drug Administration.

Cobenfy (xanomeline/trospium chloride) could bring patients what they’ve long hoped for: A means of easing the hallucinations and “voices” that disrupt their lives without the weight gain and sluggishness of current dopamine-focused drugs.

Cobenfy does affect dopamine, but indirectly through what are known as cholinergic receptors, altering the activity of another neurotransmitter, acetylcholine.

“Schizophrenia is a leading cause of disability worldwide. It is a severe, chronic mental illness that is often damaging to a person’s quality of life,” said Dr. Tiffany Farchione, director of psychiatry in the Office of Neuroscience in the FDA’s Center for Drug Evaluation and Research.

“This drug takes the first new approach to schizophrenia treatment in decades,” she said in an agency news release. “This approval offers a new alternative to the antipsychotic medications people with schizophrenia have previously been prescribed.”

According to the FDA, about 1% of people have schizophrenia, which is characterized by intrusive thoughts, hallucinations (including voices) and paranoia that can greatly interfere with daily living and social interactions with others.

It can be a debilitating illness and greatly raises the odds of dying at a young age, the agency said. Almost 5% of people with schizophrenia die by suicide, the FDA noted.

Almost all existing medications for schizophrenia have worked to bring symptoms under control by blocking dopamine receptors in brain cells.

This can render symptoms manageable, but medicated people have for decades complained of weight gain, lack of motivation and sluggishness as side effects, and quitting meds is common.

Cobenfy is made by Bristol Myers Squibb. The drug was tested in two 5-week trials which were “double-blinded”: Neither patients nor those distributing the medications knew if the patient was getting Cobenfy or a placebo.

Patient outcomes were rated on a standard 30-item scale measuring schizophrenia symptoms.

“In both studies, the participants who received Cobenfy experienced a meaningful reduction in symptoms from baseline to Week 5,” the FDA said.

Side effects — most notably gastrointestinal, such as nausea, indigestion, constipation and vomiting — could occur. A rise in heartbeat rate and acid reflux could also occur.

Because of the risk of liver damage, Cobenfy is not recommended for use by people whose livers are already impaired.

Unlike other antipsychotics used to treat schozophrenia, Cobenfy “does not have atypical antipsychotic class warnings and precautions and does not have a [FDA-mandated] boxed warning” on its label, Bristol Myers Squibb noted in a company news release.

The two trials lasted only 5 weeks, so longer-term effectiveness and safety remain unclear.

“We don’t know how it works in any substantial way past five weeks,” said Dr. David Rind, medical director of the Institute for Clinical and Economic Review, which tracks newly approved medications.

People “are a little wary of claims before they actually see what happens with the drugs,” Rind told the The New York Times. He wondered about the potential for longer term effectiveness, or neurological effects from Cobenfy, such as motor disorders.

According to the Times, Bristol Myers Squibb has said it has performed individual case studies of people who took Cobenfy for a year, which showed that they did not develop metabolic changes or any sign of movement disorder. The company said it plans to publish those results later in 2024.

Cobenfy won’t come cheap: Bristol Myers Squibb said the medicine will cost $1,850 a month, or around $22,500 a year, the Times reported.

More information

Find out more about schizophrenia and its treatment at Mental Health America.

SOURCES: Food and Drug Administration, news release, Sept. 26, 2024; The New York Times; Bristol Myers Squibb, news release, Sept. 26, 2024