Chinese Authorities Approve Narcolepsy Drug
In the world of neurological disorders, narcolepsy stands out as a rare, chronic, and debilitating condition that affects sleep-wake state stability. Characterized by excessive daytime sleepiness (EDS), cataplexy, and nocturnal sleep disturbances, the search for effective treatments has long been a priority for researchers and pharmaceutical companies.
In China, the Chinese National Medical Products Administration (NMPA) has recently approved pitolisant (Wakix) for the treatment of EDS or cataplexy in adult patients with narcolepsy. This selective histamine 3 (H3) receptor antagonist/inverse agonist, developed by RareStone Group, is now a first-line treatment therapy, as per the Chinese guidelines for diagnosis and treatment for narcolepsy (2022). Shawn Xiang, CEO of RareStone Group, expressed his pleasure at bringing a new therapeutic option for Chinese narcolepsy patients.
Besides RareStone Group, notable companies working on narcolepsy treatments in China and globally include Takeda and Centessa Pharmaceuticals.
Takeda, a major player in the field, is developing an orexin 2 receptor (OX2R) agonist named oveporexton. This drug has shown statistically significant improvements in excessive daytime sleepiness in Phase 3 clinical trials for narcolepsy type 1. Takeda is preparing to submit an FDA approval filing and expects oveporexton to become a significant treatment option. With potential sales of $1 billion by 2030 and up to $3 billion in peak yearly sales for narcolepsy type 1 alone, Takeda's development positions them as a leader in orexin-targeting therapies for narcolepsy.
Centessa Pharmaceuticals, a British-American company, is developing another OX2R agonist named ORX750 for narcolepsy types 1 and 2, as well as idiopathic hypersomnia. This drug is in clinical testing with phase 2 results pending, showing promising early data on effectiveness in promoting wakefulness.
In terms of treatment willingness, pediatric patients/caregivers rate pitolisant at 9.3, while adult patients rate it at 7.7. Pitolisant has also been recognized by the FDA as a breakthrough therapy.
It's important to note that most of the treatment drugs for narcolepsy in China are scheduled drugs with strict prescription regulations. However, pitolisant is the first approved innovative drug for narcolepsy and the only non-scheduled drug with a narcolepsy indication in China. Furthermore, all treatment drugs available in China for narcolepsy are "off-label" usage.
Despite the existing treatment options, many patients are not satisfied. RareStone Group is working with Bioprojet to bring a promising treatment drug for narcolepsy to China. Tris Pharma also has a narcolepsy treatment in its pipeline.
Unfortunately, more than half of narcolepsy patients in China have experienced misdiagnosis. The average time to diagnosis for adult patients is 2.98 years, while for pediatric/adolescent patients, it is 0.7 years.
The European Medicines Agency (EMA) and the U.S. Food and Drug Administration (FDA) have granted orphan drug designation for the treatment of narcolepsy to pitolisant (Wakix). Jazz Pharmaceuticals has also worked on a narcolepsy drug.
In conclusion, the landscape for narcolepsy treatments is evolving, with companies like Takeda, Centessa Pharmaceuticals, and RareStone Group leading the way in developing innovative therapies targeting the orexin system, a novel and promising mechanism for narcolepsy treatment.
- The search for effective treatments for narcolepsy, a rare and debilitating condition, extends beyond RareStone Group, with companies like Takeda and Centessa Pharmaceuticals also working on biotech solutions in health-and-wellness, particularly focusing on the orexin system.
- Science and technology continue to play a significant role in mental-healthcare advancement, with pharmaceuticals like pitolisant (Wakix) developed by RareStone Group addressing sleep disturbances associated with narcolepsy, improving the quality of life for patients.
- In the realm of rare diseases, the FDA and EMA have recognized pitolisant (Wakix) as a breakthrough therapy, signifying the potential of science-driven pharmaceuticals to transform mental-health management and overall health-and-wellness.
