By Yael Waknine
Medscape Medical News
February 3, 2010 — The US Food and Drug Administration (FDA) has approved collagenase clostridium histolyticum injection (Xiaflex, Auxilium Pharmaceuticals, Inc) as the first treatment for a progressive hand disease known as Dupuytren's contracture. The company expects to launch the first-in-class, orphan-designated biologic in late March 2010.
Dupuytren's is a debilitating condition caused by an abnormal buildup of collagen in the connective tissue of the palms and hands, forming thick rope-like cords that cause the fingers to curl. As the contractures increase, it may be difficult to use affected fingers to grasp objects. The condition affects an estimated 240,000 individuals across the United States and Europe and is most common among whites and men older than 50 years.
"Before the FDA approved Xiaflex, the only effective treatment for this hand disorder was surgery, which sometimes meant a long recovery and the need for physical therapy for patients," said Bob Rappaport, MD, director, Division of Anesthesiology, Analgesia, and Rheumatology of the FDA's Center for Drug Evaluation and Research, in an agency news release. "Since there are no other non-surgical alternatives for Dupuytren's contracture, Xiaflex will be an important advance in the management of this disabling condition."
The product, which contains 2 microbial collagenases derived fromClostridium histolyticum bacteria, is administered intralesionally into affected areas of the hand to soften and ultimately lyse the cords, restoring flexibility.
Its approval was based in part on data from a 306-patient study, showing that 64% of those treated with the biologic achieved finger mobility compared with 7% of those receiving placebo. In a smaller study (n = 66), the success rate was 44% (vs 5% for placebo).
Adverse events most commonly reported in the studies (incidence ? 25%) included peripheral edema, contusion, injection-site reaction, injection-site hemorrhage, and pain in the injected extremity. Although no serious allergic reactions were observed, the presence of foreign proteins poses a risk for immune response.
"With the safety and effectiveness of Xiaflex demonstrated across multiple clinical trials, physicians can now use Xiaflex to treat any symptomatic cords in patients with Dupuytren's contracture," said Larry Hurst, MD, study investigator and professor and chair, Department of Orthopedics at State University of New York Stony Brook in a company news release. "I believe that Xiaflex, as a new nonsurgical treatment, could potentially become the standard of care for Dupuytren's contracture."
Because of the potential for tendon ruptures, the FDA is requiring implementation of a risk evaluation and mitigation strategy program that will inform clinicians about how to properly perform injections and finger extensions. Access to the collagenase product will be limited to clinicians who have completed the training program, which can be obtained as a video or written manual by calling 1-877-XIAFLEX.
February 3, 2010 — 美國食品藥物管理局(FDA)核准溶組織芽胞梭菌膠原酵素注射劑(商品名Xiaflex，Auxilium Pharmaceuticals藥廠)用於一種漸進性手部疾病、Dupuytren氏孿縮症(Dupuytren's contracture)的治療，該公司預計在2010年3月底發售此類別中的第一種罕見疾病生物製劑。
這項核准是根據一個有306名病患之研究的部份資料，顯示那些以這個生物製劑治療的病患有64%達到手指可動，使用安慰劑者只有7%。在比較小型的研究中(n = 66人)，成功率為44% (安慰劑組為5%)。
Before receiving XIAFLEX, tell your healthcare provider if you have had an allergic reaction to a previous XIAFLEX injection, or have a bleeding problem or any other medical conditions. Tell your healthcare provider about all the medicines you take, including prescription and non-prescription medicines, vitamins, and herbal supplements. Be sure to tell them if you use blood thinners such as aspirin, clopidogrel (Plavix®), prasugrel hydrochloride (Effient®), or warfarin sodium (Coumadin®).