Clinical trial shows promise for fibrosis drug

NEW YORK — A drug being developed to treat a fatal scarring of the lungs has succeeded in an important clinical trial and could become the first medicine to be approved for that disease, known as idiopathic pulmonary fibrosis.

The drug, pirfenidone, slowed the decline in lung function in patients with the disease, according to an announcement Tuesday by the drug’s developer, InterMune. The company said it would apply to the Food and Drug Administration for approval of the drug early in the third quarter of this year.

InterMune Inc. shares rose $23.84, or about 170%, to $37.80 on Tuesday.


Daniel M. Rose, chief executive of the Pulmonary Fibrosis Foundation, a patient advocacy group, said the news was encouraging.

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“This disease is deadlier than 60 to 70 percent of malignancies,” he said. Pirfenidone “does not cure the disease, it does not work in every patient, but there is a cohort of patients who do receive a benefit.”

Idiopathic pulmonary fibrosis affects about 70,000 Americans, according to InterMune, although the advocacy group estimates the number could be as high as 200,000. The disease kills many people within two to five years as progressive scarring of the lungs makes it impossible to breathe.

The FDA declined to approve pirfenidone in 2010 because the drug worked in one clinical trial but not in another, nearly identical study. The agency requested that InterMune run another trial. That trial’s results were announced Tuesday.

European and Canadian regulators did approve the drug based on the earlier trials, and it is sold in those places under the name Esbriet. It has also been sold in Japan since 2008 by Shionogi Inc. under the name Pirespa.


Its reluctance to initially approve the drug could leave the FDA open to complaints that it unnecessarily delayed a treatment for desperate patients.

Rose of the Pulmonary Fibrosis Foundation, which has received contributions from InterMune, said some patients were upset and some had been ordering pirfenidone from abroad. But overall, he said, “There hasn’t been a huge amount of anger at the FDA.”

The new trial involved 555 patients in the United States and eight other countries. The main endpoint was the change in a measure of lung function called forced vital capacity.

After one year, 22.7 percent of patients who received the drug had experienced no decline in lung function, compared with only 9.7 percent of the patients who got a placebo. And only 16.5 percent of patients who got the drug had a decline of 10 percent or more in lung function or died. Among those who received a placebo, 31.8 percent had that big a decline or died.

Pirfenidone also appeared to help patients live longer. That survival benefit was not statistically significant for the new trial alone. But when the results from the new trial and the two older trials were combined, there was a statistically significant reduction in mortality.


Biogen Idec of Cambridge, Mass., and others are also testing drugs for the disease.