UK Prix Galien: First therapy for pulmonary fibrosis

The final article in our series highlighting some of the shortlisted products in the UK Prix Galien awards focuses on Esbriet, the first drug approved for idiopathic pulmonary fibrosis and the primary draw for Roche’s planned $8.3bn acquisition of its developer InterMune.

For the estimated 100,000 patients in Europe who suffer from the rare respiratory disease idiopathic pulmonary fibrosis (IPF), the approval of the first dedicated treatment in 2011 was a landmark event.

Prior to the green light given by the European Medicines Agency (EMA) for InterMune’s Esbriet (pirfenidone), the only treatment for IPF – a progressive scarring of the lungs – was aimed at reducing symptoms such as shortness of breath or cough.

For a minority of younger patients, long-term improvement can be achieved with a lung transplant. But for most patients, treatment relied on breathing oxygen through a mask and exercises to try to support lung function.

Some people manage well on that approach and remain free of symptoms for years, but others eventually go into a decline and can develop fatal complications such as heart failure, with the survival time almost impossible to predict and typically just three years from diagnosis.

Against that backdrop, Esbriet has emerged as the first approved treatment shown to slow disease progression in IPF for patients with mild-to-moderate IPF, in other words whose who still retain 50% to 80% of normal lung capacity.

In clinical trials, Esbriet – which combines anti-fibrotic activity to prevent scarring and anti-inflammatory activity – reduced the rate of disease progression by approximately 30%, limiting declines in lung function and improving exercise tolerance compared to placebo.

Meanwhile, a recently-completed pooled analysis of data from Phase 3 trials of the drug found a 52% reduction in the risk of a 10% or greater decline in percent predicted forced vital capacity (FVC) or death after a year’s treatment, suggesting its effects are long-lasting.

That level of efficacy was unprecedented in IPF and “comparable to or exceeding the effectiveness of treatments for similarly lethal diseases, such as lung cancer,” commented Rory Cameron, senior vice president and general manager for InterMune in the UK and Ireland.

Esbriet has a price tag of around $40,000-$50,000 per year in its approved markets (in addition to the EU it has also been approved in Canada) and achieved $66m in sales in the first half of this year – up from $24m in the same period of 2013.

Its sales trajectory has been held back by a delay to approval in the US, where there are another 100,000 or so eligible patients, resulting from an FDA request for more information. The company filed additional data in May and was awarded ‘breakthrough’ status by the FDA two months later, raising hopes of prompt approval.

Market research from GlobalData has suggested that the market for IPF therapies could be as much as $1.1bn across Europe and the US, which helps to explain why Swiss pharma giant Roche has offered a hefty $8.3bn to acquire InterMune and add Esbriet to its respiratory portfolio.

Despite the premium price tag, the UK’s National Institute for Health and Care Excellence (NICE) has been won over by the clinical evidence for the drug’s effectiveness and last year reversed an earlier decision preventing it from being prescribed under the National Health Service (NHS) in England and Wales.

“The fundamental goal of treatment [in IPF] is to slow disease progression, thereby prolonging survival, while optimally balancing benefit with safety and tolerability,” said Cameron.

“Esbriet is the most extensively studied and well-characterised therapy for IPF, and the only therapy with demonstrated efficacy up to 1½ years of therapy,” he added.

The eventual winner of the UK Prix Galien is scheduled to be revealed later today, 1 October. More information on all the entrants can be found here.