Rare diseases: positive results of a phase 3 study for the treatment of idiopathic hypersomnia
Idiopathic hypersomnia: results of the international phase 3 clinical trial, sponsored by Jazz Pharmaceuticals and published in Lancet Neurology, showed a clinically positive improvement in the overall severity of idiopathic hypersomnia symptoms and the patient’s self-reported global impression of change with the administration of a low-sodium calcium, magnesium, potassium and oxybate oral solution, with an overall safety profile consistent with that reported for narcolepsy
Idiopathic hypersomnia study involving 154 patients in the USA
The study, a multicentre randomised double-blind placebo-controlled trial, involved 154 adults aged 19-75 years followed at 50 sleep centres in the USA and 6 European countries between 27 November 2018 and 6 March 2020.
This evidence led to the approval in August 2021 by the US Food and Drug Administration of low-sodium oxybate as an orphan drug for the treatment of idiopathic hypersomnia in adults, and it was made commercially available in November 2021 for that indication.
RARE DISEASES? VISIT THE UNIAMO – ITALIAN FEDERATION FOR RARE DISEASES BOOTH AT EMERGENCY EXPO
What is idiopathic hypersomnia?
The term hypersomnia refers to an excessive need for sleep or a state of continuous drowsiness that interferes with everyday life.
Probable causes include conditions such as narcolepsy, sleep apnoea, restless legs syndrome, severe sleep deprivation, depression, the use of certain medications (such as tranquillisers) or the abuse of drugs and alcohol.
If the causes of hypersomnia during the day, even though there is regular sleep at night, are not known, the disorder is referred to as idiopathic or primary hypersomnia.
Idiopathic hypersomnia is a debilitating neurological sleep disorder characterised by chronic excessive daytime sleepiness, i.e. the inability to remain awake and alert during the day, resulting in an uncontrollable need to sleep or unplanned falls into sleep or drowsiness.
The main symptoms may include confusion, irritability and severe sleep inactivity or sleep drunkenness (prolonged difficulty in waking up with frequent returns to sleep).
In addition, people with idiopathic hypersomnia may experience prolonged and unrestorative night sleep, cognitive impairment in thinking and long, unrestful naps.
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