To print: Select File and then Print from your browser's menu --------------------------------------------------------------------------------------- Title: New Orphan Drug May Be Effective Cystic Fibrosis Treatment URL: http://www.pslgroup.com/dg/24EE6.htm Doctor's Guide April 30, 1997
SAN MATEO, Calif., April 30, 1997 – The U.S. Food and Drug Administration (FDA) has granting orphan drug status for SciClone Pharmaceuticals, Inc.'s CPX as a potential treatment for cystic fibrosis (CF). In addition, the company announced the start of its Phase 1 clinical trial program for CPX which will be conducted at six sites. Uniquely, this Phase 1 study will be conducted in CF patients and also measure pharmacodynamics (effectiveness). Orphan drug designation provides a company with tax credits for clinical research expenses, eligibility for a grant program to apply for funding for clinical trials, and seven years of market exclusivity to the first sponsor who receives FDA product approval for the orphan drug in the designated indication. CF is a genetic disease afflicting approximately 30,000 Americans which causes recurring respiratory infections and gastrointestinal problems. The U.S. Congress enacted the Orphan Drug Act to promote research and development of therapies for rare diseases. Cystic fibrosis and many other diseases are considered orphan diseases because they affect patient populations of less than 200,000. "Orphan drug designation by the FDA for CPX further validates our belief in the viability of pursuing CPX as a potential treatment for cystic fibrosis," said Donald Sellers, President and Chief Executive Officer of SciClone Pharmaceuticals, Inc. In January 1997, the FDA approved SciClone 's Investigational New Drug (IND) to begin clinical testing of CPX directly in CF patients rather than in healthy volunteers, thereby accelerating development of CPX by at least one year. The Cystic Fibrosis Foundation and the co-inventor of CPX for use in cystic fibrosis, Dr. Harvey Pollard of the NIH, supported SciClone in its IND filing with the FDA to gain approval to begin clinical testing of CPX directly in CF patients. "With nearly 400 CF patients dying annually, FDA approval to begin CPX clinical testing directly in CF patients meets the urgent need to accelerate the development of a potential treatment for CF that targets the root cause of the disease," said Dr. David Karlin, Vice President and Medical Director of SciClone Pharmaceuticals. "CPX, an orally available systemically acting compound, has the potential to significantly impact the management of the pulmonary and gastrointestinal manifestations of cystic fibrosis," he added. CPX is an orally available compound that targets the biochemical abnormality at the root cause of CF, the malfunctioning cystic fibrosis transmembrane conductance regulator (CFTR). In in vitro studies performed by Dr. Pollard and Dr. Kenneth Jacobson of the NIH and their research team on cultured cell lines and explanted epithelial tissue from the respiratory tracts of CF patients, CPX was shown to bind to the malfunctioning CFTR and permit it to properly perform its function, the secretion of chloride across cell membranes. CPX Clinical Trial Program Begins This Phase 1 program will include two studies. In the first study of the program, the Company will enroll 35 patients in a randomized, double-blind, placebo-controlled, ascending single oral dose study. The second study of the program will be a randomized, double-blind placebo-controlled multiple oral dose study. Both studies will evaluate the safety, pharmacokinetics and pharmacodynamics of CPX in adult patients with mild cystic fibrosis. Uniquely, each study will use nasal epithelial transmembrane potential difference and sweat chloride testing as surrogate markers to study the effectiveness of CPX. The first CPX clinical study will be conducted at six sites by the following principal investigators: Dr. Richard Aherns of the University of Iowa Hospitals and Clinics Cystic Fibrosis Center (Iowa City, IA); Dr. Moira Aitken of the University of Washington and Children's Hospital Cystic Fibrosis Center (Seattle, WA); Dr. Michael Konstan of the LeRoy Matthews Cystic Fibrosis Center, Rainbow Babies and Children's Hospital (Cleveland, OH); Dr. Richard Moss of the Stanford Cystic Fibrosis Center, Lucille Packard Children's Hospital (Palo Alto, CA); Dr. Warren Regelmann of the University of Minnesota Cystic Fibrosis Center (Minneapolis, MN); and Dr. Mark Weatherly of the Emory University Cystic Fibrosis Center, Egleston Children's Hospital (Atlanta, GA). --------------------------------------------------------------------------------------------- Copyright © 1999 P\S\L Consulting Group Inc. All rights reserved. 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