The U.S. Food and Drug Administration (FDA) has approved Xiaflex, a new drug to treat Dupuytren's contracture (or disease) that was discovered and developed by Marie A. Badalamente, Ph.D., and Lawrence C. Hurst, M.D., in the Department of Orthopaedics at Stony Brook University Medical Center. Dupuytren's contracture is a debilitating hand disorder caused by progressive accumulation of collagen that deforms fingers and limits motion and affects millions worldwide. The new treatment is the first FDA-approved non-surgical treatment for Dupuytren's contracture. Previous FDA-approved clinical trials of the drug, an injectable form of the enzyme, collagenase, showed that it significantly improves outcomes in patients with the disease.
The February 2, 2010, FDA approval comes after months of review of Xiaflex, which will be manufactured by Auxilium Pharmaceuticals, Inc., based in Malvern, Penn. Shortly after Drs. Badalamente and Hurst published their study results on the use of the collagenase treatment for Dupuytren's contracture in The New England Journal of Medicine on September 3, 2009, an FDA advisory panel had voted unanimously (12-0) to recommend the drug as a treatment to combat the hand disorder. Auxilium is moving forward to launch marketing and production of Xiaflex, which is expected to be available to orthopedic surgeons and hand specialists in March.
"The FDA decision is a monumental one in that patients with Dupuytren's contracture will now have an alternative treatment that does not require surgery and has been shown in clinical trials to be very effective," says Dr. Hurst, Co-Principal Investigator and Professor and Chair of the SBUMC Department of Orthopaedics, who indicates that hundreds of patients with the hand disorder have already contacted him about the treatment in recent weeks.
"The FDA approval is the culmination of many years of research, development and clinical trials for a treatment that is now poised to become a new standard of care for many patients with Dupuyren's contracture," says Dr. Badalamente, Professor in the SBUMC Department of Orthopaedics.
Drs. Badalamente and Hurst began their research more than 15 years ago in their laboratory when they first realized the enzyme had potential to treat Dupuytren's disease. The investigative process has resulted in a bench-to-bedside story that began with successful laboratory results on breaking up collagen within fibroproliferative tissue of the cords that cause the finger contractures. Their laboratory experiments eventually led to FDA-approved clinical trials. The FDA decision is a crowning moment in their bench-to-bedside story.
The SBUMC-led study of the use of the drug in treating 308 patients with Dupuytren's disease at 16 sites nationwide reported in the September 3, 2009, issue of The New England Journal of Medicine, indicated that the injections are safe and effective. Other participating institutions enrolling patients in the trial included Stanford Hospitals and Clinics in Palo Alto, Calif., University of California, Los Angeles, the Hospital for Special Surgery in New York, and the Indiana Hand Center in Indianapolis.
"Our study revealed that injections of the collagenase into the cords causing the finger contractures weaken the cords significantly," says Dr. Hurst. "The next day we found that we could manipulate the patient's fingers and break the cords causing the contracture. The result is frequently a complete correction of the joint contracture."