Encouraging Findings in Phase III Trials for a Narcolepsy Drug, Exhibiting Low Cardiovascular Hazards
In a significant development for those affected by narcolepsy, several new treatments are emerging, offering a fresh approach beyond the currently available Xyrem (sodium oxybate), developed by Jazz Pharmaceuticals.
Narcolepsy, a chronic sleep disorder that causes disrupted sleep cycles and excessive sleepiness during the day, has long presented a challenge for medical professionals. One of the most common symptoms is cataplexy, where sufferers experience sudden loss of muscle tone, often leading to collapse.
Jazz Pharmaceuticals' Xyrem, approved to treat cataplexy and sleepiness in narcolepsy, contains a high amount of salt, making it a potential risk factor for cardiovascular disease. However, the company's latest experimental drug boasts a lower salt content, making it more suitable for patients at risk of cardiovascular disease.
The success of this experimental drug was first announced in March 2019, with the trial showing that it was able to reduce the number of cataplexy attacks and sleepiness in narcolepsy patients compared to a placebo. The trial results were presented at the World Sleep 2019 conference in Canada.
Beyond Jazz Pharmaceuticals, the French pharmaceutical company Bioprojet SCR and its US partner Harmony Biosciences recently had a narcolepsy drug approved by the FDA. This drug, too, has a different mechanism of action to Xyrem, and, like the drugs developed by Orexia and Inexia, hasn't been shown to be addictive.
These latest treatments join a growing list of alternatives, including Solriamfetol and Pitolisant, which improve daytime alertness and reduce cataplexy via different mechanisms than sodium oxybate. AXS-12, a drug originally developed as an antidepressant but now in phase 3 trials for narcolepsy, has shown significant reductions in cataplexy frequency and improved excessive daytime sleepiness, cognition, and overall function.
Orexin receptor agonists, such as Alixorexton from Alkermes and Oveporexton, offer a targeted approach by activating orexin receptors. Both have shown positive results in clinical trials and are progressing to late-stage clinical trials.
Behavioral and lifestyle approaches continue to complement pharmacotherapy, with scheduled naps and other strategies improving daily functioning for those with narcolepsy.
These advances mark a shift toward more personalized and mechanism-specific treatments, particularly orexin agonists, which address underlying neurochemical deficits in narcolepsy. Participation in clinical trials remains an important avenue for patients seeking innovative therapies beyond currently approved options.
- The biotech industry is also actively involved in the development of new treatments for narcolepsy, as evidenced by Orexia and Inexia's non-addictive drugs that work differently from Xyrem (sodium oxybate) and improve daytime alertness and reduce cataplexy.
- In the realm of health-and-wellness and mental-health, AXS-12, originally developed as an antidepressant, has shown promising results in phase 3 trials for narcolepsy, mitigating symptoms such as cataplexy frequency, excessive daytime sleepiness, and improving cognition and overall function.
- To address the cardiovascular health-related concerns associated with current treatments like Xyrem, several pharmaceutical companies are focusing on creating drugs like Jazz Pharmaceuticals' experimental drug with lower salt content, ensuring safer and more accessible treatment options for narcolepsy patients.
