The U.S. Food and Drug Administration (FDA) has approved a new drug for schizophrenia for the first time in over 70 years, featuring an innovative mechanism of action. The capsules of Cobenfy, which combine xanomelina and trospium chloride, offer hope to thousands of patients who do not respond to existing treatments or suffer from severe side effects.

“People living with schizophrenia have essentially been treated with a single class of medications throughout their illness,” stated Ken Kramer from Karuna Therapeutics, a subsidiary of Bristol Myers Squibb, in comments to Nature News in Brief. “Now, here is a different option. This is where the greatest level of excitement comes from.”

Schizophrenia is one of the top 15 causes of disability worldwide, according to the FDA. Those affected are at a higher risk of dying young, with nearly 5 percent of them taking their own lives. The disorder causes psychotic symptoms, including hallucinations (such as hearing voices), difficulty controlling one’s thoughts, and distrust of others. It can also be associated with cognitive problems and difficulties in social interactions and motivation.

“[Schizophrenia] is a serious and chronic mental illness that often harms a person's quality of life,” declared Tiffany Farchione, director of the FDA's Division of Psychiatry.

Since the 1950s, the only available treatment options have targeted dopamine receptors in the brain, which help address symptoms of psychosis. However, these medications are not effective for all patients and can cause significant side effects, such as sedation, weight gain, and motor impairment.

The new drug, Cobenfy, targets a different group of receptors known as muscarinic acetylcholine receptors, which play a crucial role in communication within the central nervous system and have shown promise as a therapeutic target for various neurological disorders, including schizophrenia. “This schizophrenia drug adopts the first new approach in decades,” Farchione emphasized. “This approval offers a new alternative to the antipsychotic medications that have previously been prescribed to people with schizophrenia.”

However, the drug is not without side effects: between 15 and 20 percent of trial participants reported nausea and vomiting, and the prescribing information warns that it should not be used by patients with known liver impairment, urinary or gastric retention, or kidney disease.

Despite these drawbacks, the drug represents a promising alternative for millions of Americans suffering from this condition. Bristol Myers Squibb is also investigating the medication for other types of disease-related psychosis.