Promising Phase III Outcomes for Narcolepsy Treatment Showing Minimal Cardiovascular Hazards
In the ongoing quest for effective and non-addictive treatments for narcolepsy, recent developments have shown promising progress. The latest frontier in narcolepsy treatment is the development of drugs that mimic orexin, a neuropeptide deficient in narcolepsy type 1.
One of the key breakthroughs is the development of orexin receptor agonists. Takeda's oveporexton, an orexin 2 receptor (OX2R) agonist, and Alkermes' alixorexton (formerly ALKS 2680) are making headlines with their impressive results in phase 3 trials. These drugs have shown statistically significant improvements in excessive daytime sleepiness (EDS), cataplexy, attention, and quality of life, while being well-tolerated with common side effects like insomnia and urinary urgency.
Apart from orexin receptor agonists, repurposed agents are also showing promise. Axsome's AXS-12, originally an antidepressant, is undergoing phase 3 trials for narcolepsy. It has demonstrated significant reductions in cataplexy attacks, improved cognition, and overall symptoms of narcolepsy, with a good safety profile.
Non-orexin targeted options like solriamfetol and pitolisant continue to expand non-addictive treatments for narcolepsy by improving wakefulness without the abuse potential associated with traditional stimulants. Low-sodium and once-nightly oxybate formulations offer alternative dosing options that may reduce side effects and improve compliance.
Behavioral and lifestyle approaches remain important adjuncts to manage symptoms non-pharmacologically.
Among the latest developments, a new narcolepsy drug, developed by Bioprojet SCR and Harmony Biosciences, has recently been approved by the FDA. This drug, unlike the previously mentioned Xyrem, has a lower salt content and a different mechanism of action, making it a non-addictive treatment option.
Jazz Pharmaceuticals, which acquired the rights to Xyrem in 2005 when it purchased Orphan Medical, has also developed a new narcolepsy drug. The trial results of this drug were announced in March 2019, revealing that it contains only 8% of the salt found in Xyrem. The drug has shown the ability to reduce the number of cataplexy attacks and sleepiness in narcolepsy patients during a phase III trial involving 200 patients.
Orexia and Inexia are also developing alternative treatments for narcolepsy that mimic the protein orexin, which controls wakefulness.
These advancements signify a significant leap forward in the treatment of narcolepsy, offering targeted, non-addictive therapies that address the core neurochemical deficit in narcolepsy, making treatment more personalized and effective with fewer addictive risks.
Pharmaceutical companies are utilizing biotech advancements to develop new narcolepsy treatments, such as Bioprojet SCR and Harmony Biosciences' FDA-approved drug, which has a lower salt content and a different mechanism of action compared to Xyrem, making it a non-addictive treatment option.
Jazz Pharmaceuticals, through its development of a new narcolepsy drug containing only 8% of the salt found in Xyrem, demonstrates the ongoing commitment of health-and-wellness companies to providing mental-health solutions that are effective, personalized, and carry fewer addictive risks.
