Idiopathic pulmonary fibrosis (IPF) is a fatal disease in which lung tissue becomes progressively and permanently scarred. The scar tissue thickens and stiffens, making it difficult for the lungs to take in air and move oxygen into the bloodstream. IPF worsens over time, until the lungs can no longer provide enough oxygen to the body’s organs and tissues. Many patients die within three to five years of the diagnosis.
IPF, which affects about 100,000 Americans, has no known cause and no cure. And until recently, effective treatments for it were lacking. But in 2014 the U.S. Food and Drug Administration approved nintedanib (Ofev) and pirfenidone (Esbriet), the first medications shown to slow the worsening of IPF.
The drugs don’t stop the disease, and it is too soon to know how many patients they will help and whether they can extend lives. Still, these are welcome treatment options for many people with IPF. Here’s what you should know.
Effectiveness and safety
Ofev won FDA approval based on three clinical trials of 1,231 patients with IPF. Approval of Esbriet was based on the results of a large clinical trial known as ASCEND and was supported by two other trials, for a total of 1,247 IPF patients.
In the Ofev trials and ASCEND for Esbriet, patients with mild to moderate cases of IPF were randomly assigned to receive either the medication or placebo for a year. Researchers then compared the lung function of patients treated with medication versus those receiving a placebo.
Lung function was determined by forced vital capacity (FVC), a breathing test that measures how much air the lungs can hold. A decline in lung capacity indicates worsening IPF.
The findings, which were published in the New England Journal of Medicine, showed that patients treated with Ofev or Esbriet had significantly less decrease in lung function compared with the placebo groups. Each drug appeared to reduce the rate of FVC decline to a similar degree.
Studies comparing the two medications haven’t been done, so it’s uncertain whether one offers any advantages over the other. In 2015 updated guidelines, the American Thoracic Society (ATS) gave each drug a conditional recommendation without favoring one over the other.
Ofev and Esbriet are pills. Ofev slows the progression of IPF by blocking multiple pathways that may be involved in the scarring of lung tissue.
The mechanism of how Esbriet works isn’t fully understood, but the drug is believed to interfere with the production of substances in the body that promote inflammation and the formation of scar tissue.
Medication side effects
Diarrhea was the predominant side effect in the Ofev trials. Other common side effects included nausea, abdominal pain, vomiting, decreased appetite, headache, and high blood pressure.
In addition, Ofev may increase the risk of heart attack, stroke, bleeding problems, and tears in the stomach or intestinal wall. Ofev can also cause birth defects or death in an unborn baby, so women should not become pregnant while taking the drug.
The major side effects with Esbriet were nausea and increased sensitivity to sunlight that can result in a severe sunburn, rash, or skin itchiness, redness, or dryness. Patients in the Esbriet trials also reported gastrointestinal problems such as diarrhea and heartburn, upper respiratory infections, fatigue, headache, dizziness, decreased appetite, sleeplessness, and joint pain. It is not known whether Esbriet can harm an unborn baby.
Both drugs have a risk of elevated liver enzymes, which can be a sign of liver damage. Blood tests should be done before and during treatment to check how the liver is working.
Ofev and Esbriet each received a conditional recommendation from the medical society. That means that the treatment will not be appropriate for all patients. For example, some patients may not be willing to tolerate certain side effects. Another concern is that the drugs are very expensive (see below).
The guidelines also note a number of unanswered questions about Ofev and Esbriet. For instance, can they help people with severe cases of IPF? Will the drugs continue to slow the progression of IPF with treatment beyond a year? Can Ofev and Esbriet be taken in combination, or one after the other, and would that be better than taking only one of the drugs?
Although questions remain, the results so far have been promising. Many patients with IPF now have two treatments that are helping slow the course of their disease.
Help with medication costs
The wholesale price of Esbriet is about $7,800 for a month’s supply of pills, or approximately $94,000 for a year. Ofev’s wholesale cost is $8,000 per month, or about $96,000 per year.
The maker of each drug offers programs that provide free medicine for eligible patients or assistance with insurance co-pays for patients who qualify. The programs also provide nursing support. To learn more:
• For Esbriet, go to Esbriet.com or call 844-ESBRIET (1-844-372-7438) for financial assistance, or 844-693-7274 for nursing support.
• For Ofev, go to Ofev.com or call 866-OPEN DOOR (866-673-6366).
Sue Mittenthal is an award-winning independent journalist who has written about health topics for Consumer Reports, The New York Times, New York magazine, and the Choosing Wisely campaign. Her work has also appeared in Esquire, The Boston Globe, Glamour, Family Circle, Reader’s Digest, Ladies’ Home Journal, and other publications.