First novel Schizophrenia treatment in decades gains FDA approval

The U.S. Food and Drug Administration approved the drug on Thursday, greenlighting the first of a new class of treatments that could help stabilize patients’ symptoms without the uncomfortable side effects that accompany existing antipsychotics.

The new drug, called Cobenfy, works differently than the class of drugs used by many Americans today to treat schizophrenia, ones with such troublesome side effects that many people skip taking them. The Bristol-Myers Squibb drug works by mobilizing a new target in the brain that scientists made inroads researching only in the past couple of decades.

“Having a drug that doesn’t produce the classic side effects of the antipsychotic medications means that a lot of people might just take these medications," said John Krystal, a psychiatry professor at the Yale School of Medicine, who wasn’t involved in Cobenfy’s development. Krystal was an investigator in a trial studying a competing drug from AbbVie.

The treatment was the centerpiece of Bristol’s $14 billion acquisition of Karuna Therapeutics. People will be able to get the pill, taken twice a day, as soon as next month, according to Bristol Chief Commercial Officer Adam Lenkowsky. A 30-day supply will cost $1,850 before insurance and other discounts, according to the company. The price is in line with other branded oral antipsychotics, and Bristol considered the longstanding gap in drug development and accessibility in its pricing, a Bristol spokeswoman said.

Cobenfy could be a huge seller for Bristol, with some analysts forecasting more than $6 billion in peak annual sales.

Schizophrenia has long been a mystery to scientists. The chronic brain disorder can cause people to suffer delusions, hallucinations and disorganized speech. Most patients are diagnosed in their teens or before they turn 30. Schizophrenia affects about 2.8 million adults in the U.S., according to the Treatment Advocacy Center.

Existing drugs—such as Thorazine and Seroquel—help treat schizophrenia symptoms by blocking dopamine receptors, which are chemicals that the brain uses for communication. Some of the treatments have been around since the 1950s.

But regulating dopamine in the brain causes side effects such as muscle tremors, drowsiness and weight gain. Some patients also don’t respond to certain treatments, leading them to cycle through different drugs.

Cobenfy works differently. To reduce psychotic symptoms, the drug activates a pair of receptors in the brain that play a crucial role in regulating memory and other important functions of the central nervous system. The muscarinic acetylcholine receptors have been suspected by scientists to have a greater role in cognitive functions such as memory than dopamine.

Researchers serendipitously discovered the antipsychotic effects of muscarinic acetylcholine receptors 25 years ago when Cobenfy’s predecessor was in testing, according to Andrew Miller, founder of Karuna, who helped develop Cobenfy.

Cobenfy wasn’t associated in clinical trials with the common main side effects of drugs that block dopamine, according to study results, Bristol and physicians.

Cobenfy doesn’t have the same side effects as dopamine blockers because it is more targeted in the activation of acetylcholine receptors and doesn’t block dopamine. Existing drugs that block dopamine do so broadly, thereby affecting a swath of brain functions it is involved with, leading to the side effects many people experience.

“It’s a big moment in our little field," said Ken Duckworth, chief medical officer for the National Alliance on Mental Illness, an advocacy group. NAMI has received funding from Bristol. “Cognition is the holy grail of recovery," he said.

Developing novel treatments for brain disorders such as schizophrenia has vexed researchers and drug companies for years because of how complicated the brain is and because doctors can’t perform biopsies. But now, Cobenfy and other drugs like it in development are part of a resurgence in neuroscience because of scientific advancements.

“Having it on the market provides an additional option, and hopefully between pushing back on the pricing and pushing back on formulary structures, people who need it will be able to get it," said Roy Perlis, a psychiatry professor at Harvard Medical School.

Write to Jared S. Hopkins at jared.hopkins@wsj.com