Pharmaxis CF Drug Bronchitol Shortlisted for Prestigious English Orphan Drug Award

Patrícia Silva, PhD avatar

by Patrícia Silva, PhD |

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PharmaxisDrug developer Pharmaxis has announced that the company’s medication for cystic fibrosis, Bronchitol, was included in the shortlist for the UK Prix Galien Orphan Drug Award, an internationally-recognized prize that distinguishes technical, scientific, and clinical research skills necessary to develop innovative medicines.

The therapies in the competition will be analyzed by UK medical experts and evaluated according to the medical, societal, and economic benefits they are likely to provide. The judging team will be led by Professor Sir Michael Rawlins, President of the Royal Society of Medicine and former founding Chairman of the National Institute of Health and Care Excellence. The rest of the team in composed of a panel of stakeholders from the UK healthcare system.

While Pharmaxis’ therapy has been shortlisted, winning the award won’t be easy, since the 2014 Orphan Drug Award “promises to be very competitive,” according to the director for Clinical Evidence and Strategy, WG Consulting, Alison Currie. “This year has seen the highest number of orphan drug entrants – and this is something we are all delighted to see. The increase reflects the growing investment companies are making in this vital area, as well as the steady raft of national policies designed to improve health outcomes for patients with rare diseases,” she explained.

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Bronchitol was designed by Pharmaxis to reduce the amount of mucus that collects in the lungs for patients who suffer from chronic respiratory conditions, such as cystic fibrosis, bronchiectasis, and chronic bronchitis. The drug consists of a proprietary formulation of mannitol and is administered to patients as a dry powder in a hand-held inhaler. It works by hydrating the lungs, which aids in the renovation of normal lung clearance.

Clinical trials conducted by Pharmaxis confirmed the safety and efficacy of the drug in patients with cystic fibrosis and bronchiectasis. Phase 3 clinical trials demonstrated that Bronchitol is an effective drug in treating cystic fibrosis, and is being tested for the treatment of bronchiectasis as well. If approved, it will be the first targeted medication for bronchiectasis in more than 20 years. Bronchitol had already been awarded fast-track status in the U.S. and an orphan drug designation in both the US and EU.

Pharmaxis CEO Gary Philips stated that the company is “very pleased” with the nomination of Bronchitol for the Orphan Drug Award, since “to be recognized in this way highlights the potential benefits associated with the product.” Bronchitol is a drug prescribed for CF patients older than six in Australia and older than 18 in the European Union, which consists of a precision spray-dried form of mannitol into the lungs.

In addition to Bronchitol, the shortlist for the Orphan Drug Award also includes treatments for pulmonary hypertension, idiopathic pulmonary fibrosis, and multiple myeloma. All of them were launched or granted a new indication in the UK between 1st January 2012 and 31st March 2014. The results are known on October, 1 in an event at the House of Commons, London, UK.