An FDA expert panel voted 9-3 Tuesday in favor of InterMune's Esbriet (pirfenidone), a treatment for idiopathic pulmonary fibrosis. IPF is a rare and fatal lung disease that affects approximately 200,000 people in the U.S. and Europe. If approved, Esbriet would be the first treatment for U.S. IPF sufferers. The treatment has already received approval in Japan on the condition that there will be a post-marketing period during which the drug won't be widely available until further data are available.
Not all the panelists were convinced of the drug's efficacy; however, most voted that the potential benefits of the drug outweighed these concerns. "I voted yes because I've been straight down the middle the entire time. I didn't see substantive evidence of efficacy per the FDA regulations but there was clinical meaningful effect on the disease. You need to offer patients hope. If this offers a smidgen of hope, then it is worth approving," one panelist said, according to TheStreet's Adam Feuerstein. Added another, "I voted yes, opposite of my vote on the question of substantial efficacy because I don't believe there is substantial evidence of efficacy; but if I got this disease, I'd be on the next Delta flight to Japan."
The FDA doesn't have to follow the panel's recommendations, but it usually does. In a conference call, CEO Dan Welch said that if the drug is approved, it may take the company some time to ramp up production. "We chose not to make certain investments in commercial or other areas of the company until we had visibility from this meeting. So one should not expect that Esbriet would be available immediately after the approval."
During the call, analysts also attempted to suss out how InterMune would price the biologic if approved. InterMune also manufactures Actimmune, a treatment for chronic granulomatous disease that's been used off-label for the treatment of IPF. On-label, Actimmune runs in the range of $8,000 to $20,000 per year. When used off-label for IPF patients, the annual cost price per year shoots up to $50,000. "I don't know what you draw from that," noted Welch, unwilling to reveal the possible price of Esbriet. A final decision is expected May 4.
- check out InterMune's release
- read TheStreet's live blog on the panel meeting