Carles Gaig
Neurologist at the Hospital Clínic in Barcelona
This clinical trial is excellent news for patients with narcolepsy. It deserves to be widely publicised because it represents a step forward in treatment.
This type of medication (orexin B receptor agonists) looks set to be a game changer in the treatment of this disease (current treatments are symptomatic and fall short or are insufficient).
This is an excellent study, very well designed and published in a leading medical journal such as the NEJM.
A previous trial with a similar drug showed marked efficacy, but the trial was discontinued due to hepatotoxicity in some patients. This new drug does not appear to be hepatotoxic, confirming the efficacy of these drugs through their action on orexin (or hypocretin, the neurotransmitter that is lacking in the brains of people with narcolepsy). In this sense, it would be a more physiological or cause-directed treatment (giving what is missing in the brain, like giving insulin to diabetics who lack it).
[Regarding possible limitations] This is an initial, phase 2 study with only 90 patients included. Further studies are needed to further demonstrate its efficacy and confirm its safety.