Reacción a "New drug improves narcolepsy symptoms, clinical trial shows"

It is a very well designed study by the company that has investigated the product, with randomisation of participants and tested against placebo. An acceptable number of subjects has been included (ideally it would be advisable to increase the sample, but the study was methodologically complex and involved a lot of effort on the part of the participants).

It is a novelty among all current options. Previously, attempts had been made to make symptoms remit, without success, when the aim was to supplement with orexin/hypocretin (in this disease there is a deficit of this neurotransmitter). However, different products (Oveporexton is the first but not the only one) have been designed to ‘trick the brain’ by administering a type 2 receptor agonist, i.e. a drug that performs the same function as orexin/hypocretin.

This is a revolution in the approach to type 1 narcolepsy, leading to an improvement in symptoms and therefore in patients' quality of life. It is very likely that this type of drug will be the future treatment for the disease. This does not mean that other products will be abandoned; they may simply be used in certain patients or as a ‘booster’ to achieve clinical improvement.

[As for possible limitations] There are no serious adverse effects, but insomnia, increased urinary frequency, salivary hypersecretion and headache have been reported, mainly, and none of the subjects have dropped out of the study. With the modification of the molecule, no hepatotoxicity has been observed.

In any case, the studies are conducted with ‘blackleg’ patients and the real life results may show moderately different results. We look forward to its commercialisation soon so that we can offer our adult patients another alternative. And we also have families of hundreds of adolescents and children who are interested in this product, but we will have to wait until we can recruit them when the paediatric study starts.