Auxilium Pharmaceuticals, Inc. (NASDAQ: AUXL), a fully integrated specialty biopharmaceutical company, announced today that the U.S. Food and Drug Administration (FDA) has approved XIAFLEX® (collagenase clostridium histolyticum, or CCH), an in-office, biologic for the treatment of Peyronie's disease (PD). XIAFLEX is the first and only FDA-approved treatment proven effective for PD in men with a palpable plaque and a curvature deformity of 30 degrees or greater at the start of therapy.
"In my practice, treating PD has been a challenge as, until now, we have had few options to offer our patients," said Martin K. Gelbard, M.D., clinical trial investigator and clinical faculty member of UCLA School of Medicine, Department of Urology. "I believe the FDA approval of XIAFLEX is a significant achievement and offers a new option for urologists: the first approved in-office treatment to be administered non–surgically that is proven safe and effective for this physically and psychologically devastating disorder."
PD is a condition that involves the development of collagen plaque, or scar tissue, on the shaft of the penis. The scar tissue, known as a Peyronie's plaque, may harden and reduce flexibility, which may cause bending or arching of the penis during erection. PD can result in varying degrees of penile curvature deformity and disease "bother" (encompassing concern about erection appearance, erection pain and the impact of PD on intercourse and on frequency of intercourse). PD is a disease with an initial inflammatory component. This inflammatory phase is poorly understood with a somewhat variable disease course and spontaneous resolution occurring in less than 13 percent of cases. After approximately 12 months of disease, the disease is reported to often develop into a more chronic, stable phase. The incidence of PD is estimated between 3 and 9 percent; however the disease is thought to be underdiagnosed and undertreatedi. Based on U.S. historical medical claims data, it is estimated that between 65,000 and 120,000 PD patients are diagnosed every year, but only 5,000 to 6,500 PD patients are treated with injectables or surgery annually.
"Auxilium is delighted about the FDA approval of XIAFLEX for Peyronie's disease and we believe we are well prepared for commercialization of this important new indication," said Adrian Adams, CEO and President of Auxilium. "We believe that this milestone, along with other recent additions to our urology portfolio, anchors our position as a leading company in the men's healthcare area. We are proud of the strength of what is an increasingly more diversified portfolio of products, which covers treatments for low testosterone, erectile dysfunction, and now Peyronie's disease and we feel that this positions us well for future potential growth and shareholder value creation."
The FDA review and approval was based on the results of safety and efficacy data from the pivotal IMPRESS (The Investigation for Maximal Peyronie's Reduction Efficacy and Safety Studies) trials, the Phase 3 double-blinded placebo-controlled studies that assessed XIAFLEX for the treatment of PD. In IMPRESS I and IMPRESS II at 52 weeks, both co-primary endpoints met statistical significance for mean percent change in penile curvature deformity and mean change in the PDQ bother domain score for XIAFLEX subjects vs. placebo patients.
The dose of XIAFLEX is 0.58 mg per injection administered into a Peyronie's plaque. Up to eight injections (four treatment cycles) may be administered in the course of treatment. Also, a penile modeling procedure is recommended after every treatment cycle of two injections in an effort to further disrupt the plaque. If more than one plaque is present, it should be injected into the plaque causing the curvature deformity. XIAFLEX has already been approved in the U.S., EU, Canada and Australia for the treatment of adult Dupuytren's contracture (DC) patients with a palpable cord. XIAFLEX for the treatment of DC is marketed under the trade name XIAPEX® in the EU.
To support access to XIAFLEX, Auxilium has created Auxilium Advantage™ which is intended to provide a single point of contact for health care providers and patients for help accessing the product. Additionally, Auxilium worked with the FDA to develop a risk evaluation and mitigation strategy (REMS) for XIAFLEX that went into effect after the product first received FDA approval in February 2010 for adults with DC with a palpable cord. Auxilium has further collaborated with the FDA to update the REMS with an Elements to Assure Safe Use (ETASU) for XIAFLEX for the drug's use in the treatment of PD in men with a palpable plaque and curvature deformity of 30 degrees or greater at the start of therapy. The goal of the XIAFLEX REMS with an ETASU for PD is to certify that the appropriate physicians and practice sites are trained in the use of XIAFLEX and to attempt to mitigate the serious risk of penile fracture (corporal rupture) and other serious injuries to the penis such as hematoma. These serious risks are highlighted in the Boxed Warning within the Full Prescribing Information (the label).
Auxilium Pharmaceuticals, Inc.