Paget's Disease

September 5, 2005

Novartis issued positive results of a pair of clinical trials of zoledronic acid, for the treatment of Paget's disease. An NDA for the drug is currently under review by the FDA. Pooled results indicated that the drug was significantly more efficacious that the standard-of-care drug Actonel (risedronate) at 6 months, with 96% of subjects responding vs. 74% for the approved drug (p<0.001). Furthermore, zoledronic acid produced a significantly more rapid time to first response (64 days vs. 89 days from baseline; p<0.001), and maintained therapeutic response through one year in all but 1 of 113 subjects (vs. 21 of 82 for risedronate). Adverse events were similar between treatment groups; the most common overall adverse event for zoledronic acid was mild to moderate flu-like symptoms, which largely resolved within 4 days of infusion. These double-blind, active-controlled studies enrolled a combined 357 patients, who received a single 15 minute infusion of 5 mg zoledronic acid or a standard regimen of Actonel (30 mg for 60 days).