Study identifies first drug therapy for sleep apnea

Researchers from the University of California San Diego School of Medicine and their international colleagues have conducted a large-scale study demonstrating the potential of tirzepatide, known for treating type 2 diabetes, as the first effective drug for the treatment of obstructive sleep apnea (OSA), a sleep disorder characterized by repeated episodes of irregular breathing due to complete or partial blockage of the upper airway.

The findings, published online in the New England Journal of Medicine, highlight the potential of the treatment to improve the quality of life of millions of people worldwide suffering from OSA.

"This study marks a significant breakthrough in the treatment of OSA, offering a promising new therapeutic option that addresses both respiratory and metabolic issues," said Atul Malhotra, MD, lead author of the study, professor of medicine at the University of California, San Diego, and director of sleep medicine at UC San Diego Health.

OSA can lead to low oxygen levels in the blood and an increased risk of cardiovascular complications such as hypertension and heart disease. Recent research, also led by Malhotra, suggests that the number of OSA patients worldwide is approaching 936 million.

The new study was conducted in two phase III, double-blind, randomised controlled trials involving 469 clinically obese participants living with moderate to severe OSA. They were recruited from nine countries, including the US, Australia and Germany.

Participants were given or not given continuous positive airway pressure (CPAP) therapy, the most common treatment for sleep apnea, which uses a machine to keep the airway open during sleep, preventing interruptions in breathing. Patients were given either 10 or 15 mg of the drug or a placebo. The effects of tirzepatide were assessed over 52 weeks.

The researchers found that tirzepatide significantly reduced the number of interrupted breathing during sleep, a key indicator of OSA severity. This improvement was significantly greater than in the placebo group. Importantly, CPAP therapy may have become unnecessary for some participants taking the drug. The data suggest that drug therapy that targets both sleep apnea and obesity is more beneficial than treating either condition alone.

In addition, treatment with the drug improved other aspects associated with OSA, such as a reduction in cardiovascular risk factors and an improvement in body weight. The most common side effect was mild stomach problems.

“Historically, treatment for OSA has meant using sleep devices, such as a CPAP machine, to relieve breathing problems and symptoms,” Malhotra said. “However, its effectiveness depends on consistent use. This new drug treatment offers a more affordable alternative for people who cannot tolerate or comply with existing therapies. We believe that combining CPAP therapy with weight loss will be optimal for improving cardiometabolic risk and symptoms. Tirzepatide may also target specific mechanisms of sleep apnea, potentially leading to more personalized and effective treatment.”

Malhotra adds that the availability of drug therapy for OSA represents a significant advancement in the field. “It means we can offer an innovative solution that brings hope and a new standard of care to provide relief to countless individuals and their families who have struggled with the limitations of existing treatments,” Malhotra said. “This breakthrough opens the door to a new era of OSA management for people with obesity, potentially transforming the approach and treatment of this pervasive condition globally.”

Next steps include conducting clinical trials to study the long-term effects of tirzepatide.