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:
|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
|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|