NICE has confirmed its decision to reverse it opinion on Pharmaxis’ Bronchitol mannitol dry powder for the treatment of cystic fibrosis in some adults in final guidance.

The powder is recommended as an option in adults who cannot use a daily recombinant human deoxyribonuclease due to ineligibility, intolerance or inadequate response, whose lung function is rapidly declining and for whom other osmotic agents are not considered appropriate.

Bronchitol is inhaled with a hand-held, breath activated device. It has a marketing authorisation as an add-on therapy to best standard of care in adults with cystic fibrosis.

Professor Carole Longson, Health Technology Evaluation Centre Director at NICE, said the treatment is “associated with fewer unpleasant side-effects, requires less costly equipment and less time to administer than nebulised treatments.”

Cystic fibrosis currently affects around 8,000 people in the UK. It is one of the most life-threatening inherited diseases.

Although there is no cure, treatment in adults aims to clear the respiratory secretions in order to maintain lung function, as well as reducing inflammation and bacterial infection in the respiratory tract.

“Cystic fibrosis has a major impact on the quality of life of patients and their carers so we are pleased to be able to recommend a new treatment that could ease some of this burden,” said Professor Longson.

“The Committee concluded that mannitol was a good use of NHS resources when offered to some adults who can’t use other treatments, and whose lung function is rapidly declining.”

NICE original questioned “gaps and uncertainties in the evidence” of Bronchitol’s effectiveness and raised concerns about its long-term effect on lung function when it failed to recommend the treatment in draft guidance.

NICE has failed to recommend Pharmaxis’ Bronchitol in preliminary draft guidance for the treatment of cystic fibrosis (CF).

Its independent Appraisal Committee raised concerns over “gaps and uncertainties” in the evidence provided by Pharmaxis on the treatment compared with existing options.

Dr Carole Longson, Health Technology Evaluation Centre Director at NICE said the evidence supplied “did not support a positive recommendation”.

Alongside comparative concerns, the Committee also decided the analysis provided by Pharmaxis was not a true reflection of clinical practice or the efficacy of Bronchitol.

CF is one of the UK’s most common life-threatening inherited diseases that affects around 8,000 people.

The treatment, which has a marketing authorisation as an add-on therapy to best standard of care, is conveniently administered by inhalation with an inhaler. It is a proprietary dry powder form of mannitol.

The draft guidance is now open for consultation with a final decision expected in August 2012.

Pharmaxis’ cystic fibrosis (CF) treatment Bronchitol has received marketing authorisation from the European Commission.

The decision was based on two large Phase III trials where Bronchitol improved mucus clearance and lung function and reduced infectious episodes during a six month period.

Dr Alan Robertson, Pharmaxis CEO, said the authorisation is a “very significant event” allowing CF patients to “receive the proven clinical benefits of Bronchitol.”

The treatment is now available for adult patients as an add-on therapy to the best standard of care in 29 European countries.

Patients in the UK and Germany – where there is less pricing and reimbursement approval required – will be first to benefit from the approval when the treatment is officially launched at the start of June 2012.

“Pharmaxis has now secured three drug approvals in the world’s largest pharmaceutical markets: the lung function test Aridol in Europe and the US and now approval for Bronchitol in Europe and Australia,” said Dr Robertson. “This is a credit not only to the company but also the investigators and patients throughout the world who have taken part in our clinical programmes.”