Fibrosis File

Orphan Drug Status Granted

In early September, the FDA granted orphan drug status to STX-100, an investigational, humanized monoclonal antibody used in the treatment of idiopathic pulmonary fibrosis (IPF).

STX-100 targets a key pathway in the initiation and progression of pulmonary fibrosis, according to manufacturer Stromedix Inc. There are currently no FDA-approved treatments for IPF.

The drug works by targeting integrin vß6. Stromedix, which has completed a phase 1 clinical trial of STX-100, is currently planning to initiate a phase 2 clinical trial for IPF and chronic allograft nephropathy patients in 2011.

 

IPF Pirfenidone Treatment One Step Closer

Intermune Inc., is hoping to make pirfenidone available to treat IPF. Steps are being taken after the FDA recommended further research be done on the drug before they give their approval. An additional phase 3 study to demonstrate the efficacy of pirfenidone in IPF prior to marketing approval has been requested.

 

Intermune reports, “On March 23, 2010, our application to the European Medicines Agency seeking approval of pirfenidone for the treatment of IPF in adults was validated. Validation indicates that the application is complete and that the review process has begun. We anticipate submitting our responses to their questions sometime during the fourth quarter of 2010 and stand by our previous guidance of a decision regarding approvability of pirfenidone in the European Union during the first half of 2011.”

We Need Your Participation!

If you have been diagnosed with pulmonary fibrosis (PF), the Coalition for Pulmonary Fibrosis (CPF) urges you to consider being involved in clinical trials. Progress can only be made in PF by studying promising therapies and having ample patient participation in clinical trials. There is a full listing of trials on the CPF website at www.coalitionforpf.org/cpf_research_ clinical.php. The National Institutes of Health (NIH) is conducting clinical trials as part of a multi-study protocol known as IPFnet (www.ipfnet.org). The new clinical trials are testing drugs that block pathways considered key for the development of tissue fibrosis yet have, until now, never been adequately tested for their effectiveness in PF.

 

The first trial, named PANTHER, will evaluate the effectiveness of anti-oxidants. An earlier study suggested a promising role for anti-oxidants in PF. PANTHER has been adequately designed to test whether steroids and related drugs are helpful. The PANTHER- IPF: Prednisone, Azathioprine, and N-Acetylcysteine is a study that evaluates the effectiveness of N-acetyl cysteine alone and in combination with prednisone and azathioprine at preventing the loss of lung function. This trial is available to all people with IPF diagnosed in the last 48 months between 35–85 years of age with moderate disease who meet study-specific enrollment criteria.

 

“Many PF patients are treated with these types of drugs, yet the usefulness of steroids remains in question. PANTHER will answer this question once and for all, but only if patient enrollment goals are met,” said Jesse Roman, professor and chair of the Department of Medicine at University of Louisville and chair of the Education Committee in the IPFnet. “It is critical that PF patients participate in this study so that we begin to obtain answers that might lead to effective and safe treatments. Patients are crucial for the success of this research.”

 

The second trial is called ACE. The ACE trial will explore the effectiveness of blood thinners in treating PF. The use of Coumadin and related drugs is common in patients with vascular disorders, but they have not been tested extensively in the setting of PF, even though animal studies and small clinical studies suggest it may be beneficial. The ACE trial is available to all people with IPF between 35–80 years of age, regardless of time of diagnosis, who meet study-specific enrollment criteria.

To learn more information about the IPFnet trials, contact one of these clinical sites:

 

 

State	Investigator	Institution	Contact Information
Alabama	Joao de Andrade, MD	University of Alabama at Birmingham	Tonja Meadows 205-934-7630
California	Joseph Lynch, MD	University of California at Los Angeles	Eileen Callahan 310-794-8595
	Talmadge King, MD	University of California at San Francisco	Renee Jeffrey 415-476-5034
Colorado	Kevin Brown, MD	National Jewish Health	Todd Dubois 303-398-1621
Connecticut	Danielle Antin-Ozerkis, MD	Yale University School of Medicine	Jean Estrom 203-785-7324
Florida	Marilyn Glassberg, MD	University of Miami, Miller School of Medicine	Emmanuelle Simonet 305-243-3728
Illinois	Imre Noth, MD	University of Chicago	Cathy Brown 773-834-7085
Kentucky	Jesse Roman, MD	University of Louisville	Tamra Perez 502-852-1358
Louisiana	Joseph Lasky, MD	Tulane University	Sandy Ditta 504-988-4040
Michigan	Fernando Martinez, MD	University of Michigan	Debra Dahlgren 734-936-8917
Minnesota	Jay Ryu, MD	Mayo Clinic	Susan Walsh 507-293-0367
Missouri	Neil Ettinger, MD	Saint Luke’s Hospital	Sue Merli 314-576-4501
New York	Michael Kallay, MD	Highland Hospital/University of Rochester Medical Center	Elizabeth Lyda 585-233-4358
	Robert Kaner, MD	Weill Cornell Medical Center of Cornell University	Vanessa Monroy 646-962-5568
North Carolina	Lake Morrison, MD	Duke University Medical Center	Terri Haram 919-668-4562
Ohio	Jeffrey Chapman, MD	Cleveland Clinic Foundation	Susan Lubell 216-445-5872
Pennsylvania	Milton Rossman, MD	University of Pennsylvania	Susan Metzger 215-662-3115
South Carolina	Steven Sahn, MD	Medical University of South Carolina	Kimberly Argabright 843-792-3168
Tennessee	James Loyd, MD	Vanderbilt University Medical Center	Wendi Mason 615-343-7068
Texas	John Fitzgerald, MD	University of Texas Southwestern Medical Center	Barbi Estes 214-648-6729
Utah	Mary Beth Scholand, MD	University of Utah Lung Health Research Center	Laurie Brewster 801-581-5811
Washington	Ganesh Raghu, MD	University of Washington	Carolyn Spada 206-598-4967