Savient, FDA agree on phase 3 protocol for Puricase in treatment of gout

By Lisa Kerner

Erie, Pa., March 21 - Savient Pharmaceuticals Inc. said the Food and Drug Administration approves of the company's proposed phase 3 protocol, submitted as a Special Protocol Assessment, for its lead drug candidate Puricase (PEG-uricase) in the treatment of gout.

The FDA had asked for minor changes in the proposed statistical analysis of the secondary efficacy endpoints and resubmission of the Special Protocol Assessment for final approval before patient enrollment in the trial could begin.

The company anticipates filing for the orphan drug indication of the drug to control hyperuricemia in patients with symptomatic gout who cannot be treated with conventional therapy, according to a company news release.

According to Savient, the phase 3 program is designed to compare the safety and efficacy of Puricase administered by two-hour intravenous infusion every two weeks or every four weeks versus a placebo infusion.

The program consists of two replicate six-month placebo-controlled trials of about 100 randomized patients each.

Patients randomized to the placebo arms will be eligible to receive Puricase treatment in an open-label extension trial following completion of the six-month trial.

The two trials are independently powered for the primary efficacy endpoint, a responder analysis assessing patients who have normalized plasma uric acid at months three and six, the release stated.

Secondary efficacy endpoints will include an assessment of the reduction in burden of gout tophi using digital photography, reduction in the frequency of gout flares and improvement in the count of swollen and tender joints. Improvements in patient reported outcomes will be measured using the Short Form 36 (SF-36) and the Health Assessment Questionnaire-Disability Index (HAQ-DI).

"The normalization of plasma uric acid remains the key registration endpoint, as it has been for all other drugs indicated to treat gout," chief medical officer Dr. Zeb Horowitz said in the release.

"However, the Savient program will also determine the effect of Puricase (PEG-uricase) on clinical outcomes of vital importance to symptomatic gout patients, not successfully achieved in any other gout drug development program. Since the Savient study population is one for which conventional therapy has already failed or is contraindicated, the potential benefit of Puricase treatment for these patients is uniquely important."

Savient plans a phase 3 investigators meeting beginning on March 31. Patient recruitment is expected to be completed at the end of 2006 or early 2007, followed by the filing of a New Drug Application with the FDA in late 2007.

Based in East Brunswick, N.J., Savient is a specialty pharmaceuticals company that develops, manufactures and markets products that address unmet medical needs in both niche and broader markets.

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