FDA approves first new drug for schizophrenia in more than 30 years

(SILVER SPRING, Md.) — The U.S. Food and Drug Administration (FDA) on Thursday approved the first new drug to treat people with schizophrenia in more than 30 years.

Cobenfy, manufactured by Bristol Myers Squibb, combines two drugs, xanomeline and trospium chloride, and is taken as a twice-daily pill. In clinical trials, this combination helped manage symptoms such as hallucinations, delusions, and disorganized thinking, which are common in schizophrenia.

“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,” Tiffany Farchione, M.D., director of the Division of Psychiatry, Office of Neuroscience in the FDA’s Center for Drug Evaluation and Research, said in the approval announcement.

“This drug takes the first new approach to schizophrenia treatment in decades,” Farchione continued. “This approval offers a new alternative to the antipsychotic medications people with schizophrenia have previously been prescribed.”

Cobenfy offers new hope for people with schizophrenia, providing an innovative treatment option that could change how this condition is managed, according to Jelena Kunovac, MD, a board-certified psychiatrist and adjunct assistant professor at the University of Nevada, Las Vegas, in the Department of Psychiatry.

“We are overdue for a medication that targets schizophrenia with a different mechanism of action,” Kunovac told ABC News.

The first drugs for schizophrenia, including chlorpromazine and haloperidol – also known by the brand names Thorazine and Haldol, respectively – were introduced in the 1950s and revolutionized treatment of the disease. However, there have been very few new medications since then, with most subsequent FDA approvals being for variations of these older drugs.

Most schizophrenia medications, broadly known as antipsychotics, work by changing dopamine levels, a brain chemical that affects mood, motivation, and thinking, Kunovac explained. Cobenfy takes a different approach by adjusting acetylcholine, another brain chemical that aids memory, learning and attention, she said.

By focusing on acetylcholine instead of dopamine, Cobenfy may reduce schizophrenia symptoms while avoiding common side effects like weight gain, drowsiness and movement disorders, clinical trials suggest. These side effects often become so severe and unpleasant that, in some studies mirroring real-world challenges, many patients stopped treatment within 18 months of starting it.

In clinical trials, only 6% of patients stopped taking Cobenfy due to side effects, noted Dr. Samit Hirawat, chief medical officer at Bristol Myers Squibb. “That’s a significant improvement over the 20-30% seen with older antipsychotic drugs,” he added.

The most common side effects of Cobenfy are nausea, indigestion, constipation, vomiting, hypertension, abdominal pain, diarrhea, increased heart rate, dizziness and gastroesophageal reflux disease, according to the FDA announcement.

“It may prove advantageous to those who don’t tolerate what has been available,” Dr. Leslie Citrome, a clinical professor of psychiatry and expert in psychopharmacology at New York Medical College, told ABC News regarding Cobenfy. “This will provide a new approach that may work out quite nicely.”

Citrome emphasized that patients who have trouble tolerating the side effects of traditional schizophrenia medications may benefit most from Cobenfy, and that a different mechanism of action holds hope for those who have not responded adequately to existing treatments.

Schizophrenia is a mental health disorder that affects about 24 million people worldwide, or roughly one in 300 people, according to the World Health Organization (WHO). It often leads to significant challenges in daily functioning, work, and relationships, impacting both patients and their families.

The disorder typically begins in late adolescence or early adulthood and can lead to lifelong disability if not properly managed, according to the WHO, further highlighting the need for effective treatment options.

Following approval, doctors could start prescribing Cobenfy by the end of October, according to Adam Lenkowsky, executive vice president and chief commercialization officer at Bristol Myers Squibb.

Experts hope that the drug’s unique mechanism and reduced side effects will help set a new standard of care for schizophrenia patients. Studies for additional therapeutic uses, including the treatment of Alzheimer’s disease and bipolar disorder, are also underway.

Jake Goodman, MD, MBA is a psychiatry resident physician and a member of the ABC News Medical Unit.

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