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and raised concerns about its long-term effect on lung function when it failed to recommend the treatment in draft guidance.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 reversed its original decision on the use of Pharmaxis’ Bronchitol and now backs the use of the mannitol dry powder for treating cystic fibrosis in certain adult patients.

The final draft recommendation states Bronchitol may be used for patients who do not use inhaled standard treatment options, those for whom other osmotic agents are not considered appropriate and those whose lung function is rapidly declining.

Professor Carole Longson, Health Technology Evaluation Centre Director at NICE, said the positive recommendation resulted from “helpful responses” during the initial consultation period.

The previous draft guidance concluded that Bronchitol could not be recommended as a treatment option for the NHS because of “gaps and uncertainties in the evidence of its effectiveness,” Professor Longson said. Concerns were also raised about the long-term effect on lung function.

But after analysing the responses during the initial consultation period, NICE’s Appraisal Committee now concludes that the treatment is a “good use of NHS resources”.

The final draft guidance is now with consultees, who have the opportunity to appeal against NICE’s recommendation. Final guidance is expected to be published in November 2012.

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.”

Pharmaxis has received a positive opinion from the CHMP for Bronchitol to treat adults with cystic fibrosis (CF) in Europe.

The drug’s indication is to help clear mucus, improve lung function and reduce exacerbations in CF patients over the age of 18, which represents approximately two thirds of those who could benefit from the therapy.

Dr Alan Robertson, CEO of Pharmaxis, said that the positive opinion “is good news for the CF community, which has supported us throughout the development of Bronchitol”.

The treatment consists of mannitol administered as a dry powder in a hand-held inhaler and is to be used as an add-on therapy to standard medications already in use.

Professor Stuart Elborn, President of the European Cystic Fibrosis Society, said that the medication will be the first approved therapy in Europe to improve both lung function and reduce exacerbations when added to standard medications in use today.

“Life expectancy in cystic fibrosis is improving but there remains an urgent need for new therapies,” he added.

Bronchitol’s approvals in Australia and Europe are based on two Phase III clinical trials involving more than 600 CF patients.

Bronchitol was developed in Australia and will be commercialised through the EU in collaboration with the company’s marketing partner, Quintiles.

Pharmaxis plans to undertake a clinical trial in children aged 6-17, with a view to extend the drug’s licence.

Pharmaceutical company Pharmaxis researches, develops and commercialises therapeutic products for chronic respiratory disorders.