NICE Greenlights New Cystic Fibrosis Drug in UK
The UK's cost containment agency, The National Institute for Health and Clinical Excellence (NICE), has issued final guidance recommending mannitol dry powder (Bronchitol, Pharmaxis) as an option for treating cystic fibrosis in adults who can't use other treatments and whose lung function is rapidly declining.
Mannitol is a dry powder for inhalation, which has fewer unpleasant side effects, requires less costly equipment and less time to administer than nebulized treatments. The drug is indicated as an add-on therapy to improve the standard of care of adults with cystic fibrosis.
Cystic fibrosis is one of the UK's most common life-threatening inherited diseases, and currently affects around 8,000 people. Over two million people in the UK carry the faulty gene that causes cystic fibrosis-approximately 4% of the population. For a baby to be born with cystic fibrosis, both parents must be carriers of the faulty gene. If two carriers have a child, the baby has a 1 in 4 chance of having the disease.