Datamonitor: ATS 2010 - developments in IPF and cystic fibrosis

The FDA's recent refusal of Esbriet and the publication of new guidelines for the treatment of idiopathic pulmonary fibrosis drove considerable interest in this disease at the 2010 meeting of the American Thoracic Society. Furthermore, the approval of the first new inhaled antibiotic for cystic fibrosis in over a decade brought renewed focus on this rare but extremely debilitating disease.

Idiopathic pulmonary fibrosis (IPF) is an often fatal form of interstitial lung disease with no approved drug treatments in the US or EU. Unmet need is huge in this disease, which is estimated to affect 34,000 Americans per year and some studies have shown an increasing mortality rate higher than that of many cancers. A lot of injury to the lung occurs before symptoms and diagnosis and this damage is irreversible so the aim of a treatment is to try and prevent further damage. Many physicians want to know what they can do for these patients and palliative care is also very important in this group. The coverage of IPF at the 2010 American Thoracic Society (ATS) meeting highlights the threshold that this disease is at, in terms of both diagnosis and treatment.

A major event driving interest in IPF at this year's meeting was the upcoming publication of new guidelines for diagnosis and treatment of the disease. Details of the guidelines were presented, covering changes in diagnostic criteria and a thorough review of the data available on both pharmacological and non-pharmacological treatments. Guidelines were originally produced in 2000, but the latest update changes the requirements to diagnose IPF, so that if the CT scan shows clear evidence of the disease (such as a honeycombing effect indicating the fibrosis of the lung) then a biopsy is not necessary.

IPF is also at the forefront of many physicians' minds at the moment as a result of a recent surprise decision from the FDA not to approve InterMune's IPF drug pirfenidone, despite the advisory committee panel's vote in favor of approval in March. The company said the FDA sent a Complete Response Letter indicating that it requires another clinical trial to prove the drug is effective. At an InterMune-sponsored symposium at ATS 2010, Dr Ron du Bois noted that "the FDA wanted a mortality outcome, but this is very difficult in idiopathic pulmonary fibrosis clinical trials." (The primary endpoint for IPF trials tends to be the change in forced vital capacity, or FVC). Dr du Bois also announced that the company intended to continue development of this drug, which was recently approved in Japan through collaboration with Shinogi. However, on May 26, after the ATS meeting, InterMune announced the reduction of its workforce by 40%, suggesting that the company is struggling with the impact of the FDA decision.

Additional difficulties with clinical trials for IPF were also discussed at ATS 2010, including that progression varies and currently cannot be predicted so it is challenging to design clinical trials as progression may be due to the individual patient's natural history or the drug. A sub-analysis of the perfinadone data did show that patients that have a large decline in FVC show a statistically significant improvement.

The treatment of comorbidities experienced by IPF patients is one way that physicians can make these patients more comfortable. This was highlighted at ATS 2010 by data from Dr David Zisman on the use of sildenafil to treat pulmonary artery hypertension in IPF (soon to be published in the New England Journal of Medicine). It was the first randomized clinical trial in advanced IPF for any agent, and although a trend in increased FVC was seen in the sildenafil arm, it was not statistically significant. Rueful comments from physicians attending the session suggested that this was probably the most positive data that they have seen in IPF to date.

Cystic fibrosis was also a key topic at the conference, having been brought to the front of the respiratory arena by the FDA's February 2010 approval of the first new inhaled antibiotic developed for the disease in more than a decade. Cayston (aztreonam) inhalation solution was developed by Gilead and is available in a new branded device known as the Altera nebulizer system. This product joins inhaled TOBI (tobramycin solution for inhalation), which is the most widely used antibiotic treatment for cystic fibrosis.

One of the themes in this disease area at ATS 2010 was the apparent volume of therapies, and a symposium discussion titled 'Add-on or replacement?' noted that this is a new problem for physicians treating cystic fibrosis. Other talks also focused on the complexity of most treatment regimes in the disease, indicating that a simplification of these therapies is an unmet need. Adherence is a key aspect of successful treatment in most respiratory diseases, and cystic fibrosis is no different. In fact, a new collaboration between Genentech, Novartis and the Cystic Fibrosis Foundation called the iCARE study involving 700 adolescent cystic fibrosis patients shows that the industry is also willing to assist by helping to provide real-time outcome versus adherence data for patients to see the impact of non-adherence. Some preliminary results were presented at the ATS meeting, indicating the factors influencing non-adherence and misuse of devices and showing how education can assist. Physicians appeared impressed and eager to see more, suggesting a new and important avenue for industry to take note of in the treatment of this disease. Furthermore, Datamonitor believes this is a trend that could be applied to other respiratory diseases.

Datamonitor: Stakeholder Opinions: Respiratory viral infections - Respiratory syncytial virus, parainfluenza virus and human metapneumovirus as promising new targets:
http://www.reports-research.com/studien/stakeholder-opinions-respiratory-viral-infections-respiratory-syncytial-virus-parainfluenza-virus-human-metapneumovirus-promising-targets-p-76125.html

Datamonitor: Launch Strategies: Analyzing Market Trends and Regulatory Impacts:
http://www.reports-research.com/studien/launch-strategies-analyzing-market-trends-regulatory-impacts-p-14155.html

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