By ANDREW POLLACK The New York Times
The Food and Drug Administration on Tuesday declined to approve a drug from InterMune that would have been the first treatment for a fatal lung disease that afflicts about 100,000 Americans.
InterMune said in a press release that the agency had requested another clinical trial to prove that the drug, called pirfenidone, was effective against the disease, known as idiopathic pulmonary fibrosis.
InterMune’s stock price plunged about 80 percent in after-hours trading to $9.47, after finishing regular trading at $45.44. The stock had tripled since early March, when an advisory committee to the F.D.A. voted 9 to 3 to recommend approval of the drug.
The company said it would meet with the F.D.A. as soon as possible to discuss the drug, which it had planned to sell under the brand name Esbriet and which analysts had predicted could achieve annual sales of $1 billion or more.
The data on the drug’s effectiveness posed a dilemma for both the advisory committee and the F.D.A. itself because the drug worked in one clinical trial but not in a second. The F.D.A. usually requires two positive clinical trials for approval.
The agency also questioned whether the benefit provided by the drug even in the successful trial was meaningful. The drug cannot repair damage already done to the lungs and make patients better.
But it did seem, at least in one trial, to modestly slow the decline of lung function compared to a placebo. It did not prolong patients’ lives by a statistically significant amount, though the trial was too small to have detected such an effect unless it were huge.
But some committee members said that leniency was needed because there were no other treatments approved for idiopathic pulmonary fibrosis. About 15 patients or family members of patients testified in favor of approval.
The disease is a progressive scarring of the lungs with an unknown cause that is almost always fatal, with a median survival of about three to five years.
“They suffocate from their lungs filling up with Jell-O,” Dr. Paul W. Noble, an expert on the disease at Duke University, told the advisory committee meeting.
Most people affected are over 50, and it is not clear how many people have the disease. A typical number used is about 100,000 Americans, with 30,000 new cases a year. Experts say the number of cases appears to be increasing, which might be the result of better diagnosis or of the aging of society.
A lung transplant can extend lives but there are a limited number of donor lungs available.
Mary Lou Rocha, who has the disease, said she was disappointed by the F.D.A.’s decision.
“People such as myself that have this terrible disease, we have no hope anywhere other than a lung transplant,” she said Tuesday. “I know myself and a lot of other people were depending on this.”
Ms. Rocha, who is 71 and lives in Cypress, Calif., said she could no longer bowl and ride her bicycle because of coughing and shortness of breath. She is also starting to have trouble walking up the stairs in her home.
Pirfenidone is approved in Japan, where it is sold by Shionogi. Analysts held out some hope that data from Shionogi’s trial might provide the extra evidence the F.D.A. wants, without the need for InterMune to spend years doing another trial.
InterMune, based in Brisbane, Calif., had a previous setback when a different drug, called Actimmune, failed to extend the lives of patients with pulmonary fibrosis. The company in 2006 paid $37 million to settle federal charges that it had marketed Actimmune off-label for pulmonary fibrosis.
Other drugs that have failed in clinical trials as treatments for the disease include the cancer wonder drug Gleevec, the arthritis blockbuster Enbrel and a drug for pulmonary hypertension known as Tracleer (Bosentan). Results are expected soon from a trial testing Pfizer’s Sildenafil, which is approved to treat pulmonary hypertension but is better known as Viagra.
“You Have the Power to Save Lives – Register to be an organ and tissue donor & Tell Your Loved Ones of Your Decision”
Register to be a donor in Ontario or Download Donor Cards from Trillium Gift of Life Network. NEW for Ontario: recycleMe.org - Learn The Ins & Outs Of Organ And Tissue Donation. Register Today! For other Canadian provinces click here
In the United States, be sure to find out how to register in your state at ShareYourLife.org or Download Donor Cards from OrganDonor.Gov
In Great Britain, register at NHS Organ Donor Register
In Australia, register at Australian Organ Donor Register
Your generosity can save up to eight lives with heart, kidneys, liver, lungs, pancreas and small intestine transplants (see allotransplantation). One tissue donor can help 75 to 100 other people by donating skin, corneas, bone, tendon, ligaments and heart valves
Has your life been saved by an organ transplant? "Pay it forward" and help spread the word about the need for organ donation - In the U.S. another person is added to the national transplant waiting list every 11 minutes and 18 people die each day waiting for an organ or tissue transplant. Organs can save lives, corneas renew vision, and tissue may help to restore someone's ability to walk, run or move freely without pain. Life Begins with You