Noqdirna, the only licenced treatment for the symptomatic treatment of nocturia due to idiopathic nocturnal polyuria in adults including the over 65s has been launched, following approval by the UK Medicines and Healthcare Products Regulatory Agency, Ferring Pharmaceuticals has announced.
Nocturia – the need to wake at night to pass urine – is a complex medical condition, impacting around 8.63 million people in the UK. Night-time overproduction of urine, or nocturnal polyuria, is thought to contribute in up to 76–88% of these cases.
The condition can result in an increase in falls and fractures, due to the need for night-time bathroom trips or due to sleepiness during the day. In addition, poor sleep associated with nocturia can impact physical, mental and emotional health in adults of all ages, leading to reduced daytime productivity, a decreased quality of life and an increased morbidity and mortality – studies have shown that waking up two or more times a night to urinate increases a person’s risk of mortality by up to 22%.
The burden to the UK economy is estimated to be £1.35 billion a year through hospital costs of managing nocturia and its associated consequences, and £4.32 billion a year through work absenteeism and loss of productivity.
Steve Howson, Ferring UK’s General Manager, said, “We are delighted that Noqdirna can now be made available to patients in the UK. This is a significant milestone in making sure patients of all ages, including those over 65 years old, have a treatment option to help reduce the burden of their condition. Ferring Pharmaceuticals remains committed to improving the diagnosis and treatment of nocturia, and to ensuring that patients continue to benefit from these treatment advances.”
Developed to reduce the amount of urine the kidneys produce, Noqdirna once-daily lyophilisate tablets are administered sublingually without the need for water in gender-specific, low doses, tailored specifically for men (50 µg) and women (25 µg).
The approval of Noqdirna is based on two Phase III studies (CS40 and CS41), with CS40 investigating the efficacy and safety of 25 µg in adult women and CS41 investigating the efficacy and safety of 50 µg in adult men. Both trials demonstrated that the treatment significantly reduced the average number of night-time urinations, compared with the placebo groups. Noqdirna was shown to nearly double the probability of patients achieving the primary endpoint of the studies – listed as a reduction of night-time voids by 33%.
Noqdirna was also shown to reduce nocturnal urine volume in men and women by more than 200ml, and increase time to first void in people with nocturia, allowing people to have an average undisturbed sleep period of about 4.5 hours. This extended undisturbed sleep is important due to the early hours of sleep making up the majority of the night’s deep sleep – interruption of which is associated with a range of health issues.
Overall, the drug was well tolerated with the most common adverse events being dry mouth, nausea, fatigue and headache which occurred at ≥5%. There was no incidence of severe hyponatraemia in CS40 and only two cases of severe hyponatraemia occurred in patients in CS41 who took the 50 µg dose (≤1%).
James Green, Urology Network Lead at Barts Health and Visiting Professor at London South Bank University said: “Nocturia is a worrying problem for patients and their partners. Constantly getting up at night to pass urine has a major impact on patient’s quality of life, and in the elderly it increases the risk of trips and falls. Having a new formulation that can help with this will make a real difference to thousands of patients.”
Oral desmopressin has previously been available in higher doses for other indications, however this approval came with an upper age limitation of 65 years. Trials with Noqdirna have documented the treatment of lower, gender-specific doses to be effective and well tolerated in adults of all ages, though sodium monitoring in adults over 65 years of age is warranted.
Jonathan Rees, a GP at Tyntesfield Medical Group, North Somerset, and Founder of the Primary Care Urology Society welcomed the news. “Nocturia is a very common symptom encountered in primary care and can be very difficult to treat effectively,” he said. “We know that nocturia can have a significant impact on quality of life, and also increased risk of falls and fractures. Nocturnal polyuria often does not respond to lifestyle changes or medication currently available, so the addition of Noqdirna to our armoury is highly welcome, particularly as we can now use this in patients over 65 unlike existing desmopressin formulations.”