Rare Disease Report

New Idiopathic Pulmonary Fibrosis Drug Granted Orphan Drug Designation

APRIL 10, 2015
Christina T. Loguidice

Idiopathic pulmonary fibrosis (IPF) is a chronic, progressive fibrosing interstitial pneumonia of unknown etiology. The condition has a poor prognosis, with a median survival rate between 2.5 to 3.5 years, and it most commonly affects older adults (median age, 66 years), particularly those with a history of cigarette smoking.[1] Historically, treatment involved managing comorbidities and taking measures to prevent respiratory infections (eg, administering influenza and pneumococcal vaccinations), but in late 2014, the US Food and Drug Administration (FDA) approved two drugs that block several of the pathways thought to cause pulmonary fibrosis: nintedanib (Ofev) and pirfenidone (Esbriet).[2,3] Both agents were approved through a process that involved fast-track, priority review, orphan product, and breakthrough designations. More recently, in February 2015, the FDA granted orphan drug designation status to a new antifibrotic drug: PBI-4050.[4] Animal studies have thus far shown PBI-4050 to compare favorably to both previously approved antifibrotic agents.[4]
In animal models emulating pulmonary fibrosis in humans, PBI-4050 was found to significantly reduce tissue scarring in the lungs, indicating its potential for improving and stabilizing lung function. In addition, when it was combined with another approved antifibrotic drug, there was significant reduction of fibrotic markers, suggesting a synergistic clinical benefit.
“We are pleased to have secured an orphan drug designation for our lead drug candidate for the treatment of this devastating disease,” said Pierre Laurin, president and CEO of ProMetic, in a company news release.[4] “This designation supports our decision to aggressively pursue the development of PBI-4050 in various fibrosis-related medical conditions,” he added.
Several trials are already set to launch in Canada, where PBI-4050 was recently cleared to commence clinical trials in patients with IPF.[5] A phase 1 clinical trial of PBI-4050 that included 40 healthy volunteers found the agent to be safe and well tolerated, with no serious adverse events reported. This has led ProMetic to proceed with a 12-week, open-label, single-arm, exploratory, phase 2 study that will evaluate the safety and tolerability of PBI-4050 in 40 patients with IPF. In this trial, the effects of PBI-4050 on pulmonary function, disease progression, and inflammatory and fibrotic markers will be closely examined.[5]
ProMetic is also evaluating PBI-4050 for patients with chronic kidney disease (CKD), and it has started enrolling patients with CKD in the multidose part of a phase 1b trial.[5]  Results from this trial are expected by the end of the first quarter of 2015.


1. Ley B, Collard HR, King TE Jr. Clinical course and prediction of survival in idiopathic pulmonary fibrosis. Am J Respir Crit Care Med. 2011;183(4):431-440.
2. FDA approves Ofev to treat idiopathic pulmonary fibrosis. U.S. Food and Drug Administration [press release]. Bethesda, MD; US Food and Drug Administration; October 15, 2014. http://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm418994.htm. Accessed April 10, 2015.
3. FDA approves Esbriet to treat idiopathic pulmonary fibrosis [press release]. Bethesda, MD; US Food and Drug Administration; October 15, 2014.  http://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm418991.htm. Accessed April 10, 2015.
4. US FDA grants orphan drug designation to ProMetic’s PBI-4050 drug for the treatment of idiopathic pulmonary fibrosis [press release]. Laval, Quebec, Canada; ProMetic; February 17, 2015. http://www.prometic.com/en/news-events/press-release-us-fda-grants-orphan-drug-887.php?y=2015. Accessed April 10, 2015.
5. ProMetic’s PBI-4050 cleared to commence clinical trials in patients with idiopathic pulmonary fibrosis [press release]. Laval, Quebec, Canada; ProMetic; February 4, 2015. http://www.prometic.com/en/news-events/press-release-prometics-pbi-4050-cleared-commence-clinical-883.php?y=2015. Accessed April 10, 2015.

Rare Disease Pearls: Idiopathic Pulmonary Fibrosis Survival

  • Men are more likely to develop IPF, but sex differences in survival have been inconsistent.
  • Factors associated with shortened survival in patients with IPF include older age, smoking history, lower body mass index, more severe physiologic impairment, greater radiologic extent of disease, and the development of other complications or conditions (eg, pulmonary hypertension, emphysema, bronchogenic cancer).
  • The highest death rates occur in the winter, even when excluding infections.
  • Several biomarkers obtained from blood and bronchoalveolar lavage fluid have correlated with disease progression and survival in IPF; for example, B-type natriuretic peptide was shown to be a better predictor of survival than echocardiographic assessment of pulmonary hypertension.
            Source: Am J Respir Crit Care Med. 2011;183(4):431-440.
A chest radiograph of a patient with Idiopathic Pulmonary Fibrosis (IPF) called "Radiological evaluation through HRCT" by IPFeditor - Own work. Licensed under CC BY-SA 3.0 via Wikimedia Commons - http://commons.wikimedia.org/wiki/File:Radiological_evaluation_through_HRCT.jpg#/media/File:Radiological_evaluation_through_HRCT.jpg

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