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.
A sign for the Food And Drug Administration is seen outside of the headquarters on July 20, 2020 in White Oak, Md.
Sarah Silbiger/Getty Images, FILE
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.
Schizophrenia is a chronic and severe mental disorder that affects how a person thinks, feels, and behaves. It is estimated that approximately 1% of the global population suffers from schizophrenia, making it one of the most common mental illnesses worldwide. Despite its prevalence, treatment options for schizophrenia have remained relatively unchanged for the past few decades. However, a recent breakthrough in the field of psychiatry has brought hope to those suffering from this debilitating condition.
The U.S. Food and Drug Administration (FDA) recently approved the first new drug for schizophrenia in over 30 years. The drug, known as lumateperone and marketed under the brand name Caplyta, is a novel antipsychotic medication that offers a new treatment option for individuals with schizophrenia.
Lumateperone works by targeting multiple neurotransmitter receptors in the brain, including serotonin, dopamine, and glutamate receptors. This unique mechanism of action sets it apart from traditional antipsychotic medications, which primarily target dopamine receptors. Clinical trials have shown that lumateperone is effective in reducing the symptoms of schizophrenia, such as hallucinations, delusions, and disorganized thinking.
One of the key advantages of lumateperone is its favorable side effect profile. Traditional antipsychotic medications are often associated with significant side effects, such as weight gain, metabolic abnormalities, and movement disorders. In contrast, lumateperone has been shown to have a lower risk of these adverse effects, making it a more tolerable option for patients.
The approval of lumateperone represents a significant milestone in the treatment of schizophrenia. For the first time in over three decades, individuals with schizophrenia have access to a new medication that offers both efficacy and tolerability. This breakthrough has the potential to improve the quality of life for millions of people living with this challenging mental illness.
In addition to its efficacy in treating schizophrenia, lumateperone may also have potential applications in other psychiatric disorders. Clinical trials are currently underway to investigate its effectiveness in treating bipolar disorder and major depressive disorder. If successful, lumateperone could become a valuable tool in the treatment of a wide range of mental health conditions.
Overall, the approval of lumateperone by the FDA represents a significant advancement in the field of psychiatry. This new drug offers hope to individuals with schizophrenia and provides clinicians with a valuable treatment option for managing this complex and often debilitating condition. As researchers continue to explore the potential benefits of lumateperone, the future looks promising for those affected by schizophrenia and other psychiatric disorders.
