UK's NICE reverses earlier negative decision on Pharmaxis' CF treatment Bronchitol

Australia’s Pharmaxis (ASX: PXS) says that the UK drugs watchdog the National Institute for Health and Clinical Excellence (NICE) has issued a positive recommendation in its Final Appraisal Determination (FAD) for Bronchitol (mannitol dry powder for inhalation), licensed for the treatment of adult cystic fibrosis (CF) patients aged 18 years and above as an add-on therapy to best standard of care. In doing so, the NICE has reversed an earlier decision not to recommend National Health Service use of Bronchitol (The Pharma Letter June 1).

According to the European unit of Pharmaxis, Bronchitol is the first CF product to have been fully reviewed and received a positive recommendation from NICE. The recommendation provides a new option for CF patients critically requiring new treatments and who have the highest unmet medical need. This includes those patients who cannot use rhDNase (recombinant human deoxyribonuclease) because of ineligibility, intolerance or inadequate response, whose lung function is rapidly declining (forced expiratory volume in 1 second [FEV1] with a decline greater than 2% annually) and for whom other osmotic agents are not considered appropriate.

“The clinical studies of Bronchitol showed very promising results for what is a complex therapeutic area,” said Diana Bilton, director of the Adult Cystic Fibrosis Unit at the Royal Brompton Hospital, London, and lead investigator for the first of the Bronchitol Phase III trials, quoted by the drugmaker. “This new treatment has the potential to delay lung function decline in CF patients, thereby reducing the associated risks of exacerbation and mortality. Bronchitol brings a clear step change in efficacy and ease of use to a patient population that currently spend large amounts of time on laborious nebulised therapies. These patients will be delighted to have a new inhaled treatment option,” Dr Bilton added.