BioSpecifics Technologies Corp., a biopharmaceutical company developing first in class collagenase-based products marketed as Xiaflex in the US, has announced that its partner Auxilium Pharmaceuticals, Inc. (Auxilium) submitted a supplemental Biologics License Application (sBLA) to the US Food and Drug Administration (FDA) for Xiaflex for the potential treatment of Peyronie’s disease.
If approved by the FDA, Xiaflex is expected to be the first and only biologic therapy indicated for the treatment of Peyronie’s disease, an excess of inelastic collagen causing penile curvature deformity. Xiaflex is currently approved in the US, European Union and Canada for the treatment of adult Dupuytren’s contracture patients with a palpable cord.
“We are very pleased to announce Auxilium’s sBLA filing to the FDA for Xiaflex for Peyronie’s disease and believe that, if approved, Xiaflex will provide a significant therapeutic advantage for Peyronie’s disease patients who currently have no FDA-approved minimally invasive treatment options for this physically and psychologically devastating condition,” stated Thomas L. Wegman, president of BioSpecifics. “We look forward to receiving the potential FDA approval of Xiaflex for Peyronie’s disease by the end of 2013.”
Auxilium has requested Priority Review designation for this sBLA submission and the company expects to hear back from the FDA on Priority Review designation within approximately 60 days from the filing date. If granted, the FDA’s goal for completing a Priority Review, per Prescription Drug User Fee Act guidelines, is six months from date of receipt. Priority Review may be granted to drugs that offer major advances in treatment, or provide a treatment where none exists. Xiaflex was granted orphan drug designation in the US by the FDA in January 1996 for this indication, which will allow for 7 years of protection, if approved.
Auxilium’s sBLA submission is based on data from the company’s IMPRESS (The Investigation for Maximal Peyronie's Reduction Efficacy and Safety Studies) phase III clinical program and other controlled and uncontrolled clinical studies, in which over 1,000 Peyronie’s disease patients were enrolled and received over 7,400 injections of Xiaflex.
Results from Auxilium’s two randomized, double-blind, placebo-controlled studies at 52 weeks demonstrated statistically significant improvements in both co-primary endpoints of the trials, including penile curvature deformity and patient-reported bother compared to placebo. Data from the IMPRESS I trial met statistical significance with a 37.6% mean reduction in penile curvature deformity for Xiaflex subjects (p=0.0005) and a 3.3 point improvement in the Peyronie’s Disease Questionnaire (PDQ) bother domain for Xiaflex subjects (p=0.0451). Data from the IMPRESS II trial met statistical significance with a 30.5% mean improvement in penile curvature deformity for Xiaflex subjects (p=0.0059) and a 2.4 point improvement in the PDQ bother domain for XIAFLEX subjects (p=0.0496).
Xiaflex was generally well-tolerated. The most common treatment related adverse events reported in the Phase III studies were local to the treatment site and consistent with adverse events reported in previous Peyronie’s disease trials with Xiaflex, which included injection site haematoma, pain and swelling. Serious adverse events included corporal rupture (penile fracture) in 3 subjects in the placebo controlled studies.
Peyronie's disease is characterized by the presence of inelastic collagen on the shaft of the penis, which can cause the penis to curve during erection, and may make sexual intercourse difficult or impossible in advanced cases. Significant psychological distress is common among sexually active patients with Peyronie's disease. Currently, there are no FDA-approved pharmaceutical therapies for this condition.