Study identifies first drug treatment for sleep apnea

Researchers at the University of California, San Diego School of Medicine and international collaborators led a pioneering global study demonstrating that tirzepatide, known for its effectiveness in managing type 2 diabetes, may be the first effective drug treatment for obstructive sleep apnea (OSA), a sleep-related disorder caused by complete or partial blockage of the upper airway, resulting in repeated irregular breathing.

The results will be published online on June 21, 2024. New England Journal of Medicinehighlights the potential of the treatment to improve the quality of life for millions of people worldwide who suffer from OSA.

“This study marks an important milestone in the treatment of OSA, providing a promising new treatment option that addresses both the respiratory and metabolic complications,” said Atul Malhotra, MD, lead author of the study, professor of medicine at UC San Diego and chief of the Department of Sleep Medicine at UC San Diego Health.

OSA can cause low oxygen levels in the blood, which can lead to an increased risk of cardiovascular complications such as high blood pressure and heart disease. Recent research, also led by Malhotra, suggests that the number of people suffering from OSA worldwide could be close to 936 million.

Conducted in two Phase III double-blind randomized controlled trials, the new study cohort included 469 participants diagnosed with clinical obesity and moderate to severe OSA. Participants were recruited from sites in nine countries, including the United States, Australia, and Germany. Participants either used or did not use continuous positive airway pressure (CPAP) therapy, the most common sleep apnea treatment that uses a machine to keep the airway open while sleeping to prevent interruptions to breathing. Patients received 10 mg or 15 mg of the drug via injection, or a placebo. The effects of tirzepatide were evaluated over a 52-week period.

The researchers found that tirzepatide led to a significant reduction in the number of breathing interruptions during sleep, a key metric used to measure the severity of OSA. This improvement was much greater than that seen in participants who received a placebo. Importantly, some participants who took the drug reached a level where they may no longer need CPAP therapy. Substantial data suggests that pharmacotherapy targeting both sleep apnea and obesity may be more beneficial than treating either one alone.

In addition, medication improved other aspects related to OSA, such as reducing cardiovascular risk factors and improving weight. The most common side effect reported was mild stomach problems.

“Historically, treating OSA has meant using a device like a CPAP machine while sleeping to relieve breathing difficulties and symptoms,” said Malhotra. “But its effectiveness relies on continued use. This new drug treatment offers a more accessible alternative for those who cannot tolerate or adhere to existing treatments. We believe that the combination of CPAP therapy and weight loss is optimal for improving cardiometabolic risk and symptoms. Tirzepatide may also target specific underlying mechanisms of sleep apnea, which may lead to more personalized and effective treatments.”

Malhotra added that the availability of pharmacological treatment for OSA is a major advancement in the field.

“This means we can deliver an innovative solution that brings hope and a new standard of care to countless people and their families who suffer from the limitations of existing treatments,” said Malhotra. “This breakthrough opens the door to a new era of OSA management for people diagnosed with obesity and has the potential to transform how this pervasive disease is addressed and treated globally.”

The next steps include conducting clinical trials to examine tirzepatide's long-term effects.

Co-authors on the study include Ronald Grunstein of the University of Sydney, Ingo Fitze of the University Hospital Berlin, Teri Weaver of the University of Illinois at Chicago, Susan Redline, Ali Azarbagin and Scott Sands of Harvard Medical School, Richard Schwab of the University of Pennsylvania, Julia Dunn, Sujatro Chakradhar, Matthijs Bank and Josef Bednarik of Eli Lilly and Company.

Funding for this study was provided in part by Eli Lilly and Company.

Disclosure: Atul Malhotra serves as a consultant for Eli Lilly and Company.