FDA Approves First Non-Surgical Therapy for Peyronie's Disease

The US Food and Drug Administration (FDA) has approved Xiaflex (collagenase clostridium histolyticum) as the first non-surgical treatment for Peyronie’s disease, a physically and psychologically distressing penile deformity estimated to affect between 65,000 and 120,000 male patients annually.

Initially approved in 2010 to treat patients with a progressive hand disease known as Dupuytren's contracture, the injectable biologic drug marketed by Auxilium Pharmaceuticals, Inc., is now believed to break down built-up scar tissue in the penis that causes an abnormal bend upon erection, which can lead to pain during sexual intercourse and other bothersome issues.

Xiaflex’s new indication was based on results from the phase 3 Investigation for Maximal Peyronie’s Reduction Efficacy and Safety Studies (IMPRESS). In those trials, 832 men with a curvature deformity of at least 30 degrees were randomized to receive 4 treatment cycles of Xiaflex — each consisting of 2 0.58 mg penile injections and 1 penile modeling procedure performed by a physician — or placebo for 52 weeks. At the conclusion of the studies, Xiaflex was shown to “significantly reduce penile curvature deformity and related bothersome effects compared with placebo,” the FDA said.

Audrey Gassman, MD, deputy director of the Division of Bone, Reproductive and Urologic Products in the FDA’s Center for Drug Evaluation and Research said in a press release that the approval “expands the available treatment options for men experiencing Peyronie’s disease and enables them, in consultation with their doctor, to choose the most appropriate treatment option.” That’s good news for urologists like Martin K. Gelbard, MD, who noted in a seperate announcement that “treating Peyronie’s disease has been a challenge as, until now, we have had few options to offer our patients.”

However, Xiaflex for the treatment of Peyronie’s disease is available only through a restricted program under a Risk Evaluation and Mitigation Strategy (REMS) that requires healthcare professionals experienced in treating male urological diseases to enroll and complete training in administering the drug, as it may potentially cause penile fracture and other serious penile injury. According to the FDA, the most common adverse events reported in the IMPRESS trials included penile hematoma, penile swelling, and penile pain.