The UK’s drugs watchdog has given another cystic fibrosis drug the thumbs up for NHS use this week – this time for Pharmaxis’ Bronchitol (mannitol dry power).
The final draft recommendation, which allows treatment in adults whose lung function is rapidly declining and who do not use inhaled mucolytics for certain reasons, follows the green light from the National Institute for Health and Clinical Excellence given to Novartis’ Tobi Podhaler (tobramycin inhalation powder) earlier this week.
Bronchitol is inhaled with a hand-held, breathe activated device. It acts by inducing an influx of water into the airway, improving hydration of airway secretions, and increasing mucus clearance by making it less thick and sticky and stimulating a cough.
The drug had previously not been recommended by NICE because of gaps and uncertainties in the evidence on its effectiveness compared with other treatments used in the UK. There was also some concern about the long-term effect of the drug on lung function.
However, Professor Carole Longson, health technology evaluation centre director at NICE, said the U-turn followed discussions with patient experts and clinical specialists who said Bronchitol, as a dry powder inhalation product, could ease the burden of living with cystic fibrosis. It was also associated with fewer side effects, was less costly and required less time to administer than nebulised treatments.
“Following the helpful responses received during consultation on the draft guidance, the Committee concluded that mannitol was a good use of NHS resources when treatment was offered to those who cannot use hypertonic saline or rhDNase and whose lung function is rapidly declining.”
The Cystic Fibrosis Trust has welcomed the positive decision, saying the drug is a key treatment that helps to improve the quality of life for people with cystic fibrosis, by reducing the burden of care.
Jo Osmond, director of clinical care at the CF Trust said: “This treatment is a step change in terms of improving quality of life and significantly improving lung function for people with CF, specifically because mannitol is a dry powder inhaled treatment used to aid lung clearance.”
Meanwhile, efforts continue to make the cystic fibrosis drug Kalydeco (ivacaftor) available on the NHS after the drug was denied on the grounds of cost. More than 12,000 people have signed a petition, organised by the Cystic Fibrosis Trust, which has been presented to the manufacturer Vertex requesting it to drop the drug’s price of £182,000 a year per patient. Kalydeco works in patients that have the specific G55ID gene mutation, which affects about 440 cystic fibrosis patients in the UK.