Savient Resubmits Gout BLA

Savient Pharmaceuticals has resubmitted its BLA to the FDA for Krystexxa as a treatment for chronic gout in patients refractory to conventional therapy. The submission received a complete response letter (CRL) in July 2009.

The resubmission includes data from three consecutive manufacturing validation batches of the pegloticase API, additional and improved analytical methods for the control and release of pegloticase API and Krystexxa and other data which are designed to address the issues in the letter. It also includes a Safety Update from the remaining studies that were ongoing at the time of the previous 120-Day Safety Update. Additional documents included in the resubmission are proposals for product labeling and prescribing information, REMS program materials and a patient Medication Guide, all of which were requested in the CRL.

“We believe that this resubmission combined with the submissions made and planned by our third party contract manufacturer fully addresses the deficiencies and observations raised and provides the additional materials requested in the CRL that we received from the FDA on July 31, 2009, which were further clarified in our meeting with the FDA on September 14, 2009,” stated Paul Hamelin, president of Savient. “We look forward to the advancement of this novel biologic through the regulatory review process including the potential reinspection of our contract manufacturer in the months ahead. We continue to believe that Krystexxa has the potential to fill a void in an area of significant unmet medical need and are confident that this is a quality resubmission package that can support a favorable determination by the FDA.”

Sigma-Tau PharmaSource (formerly Enzon) is the contract manufacturer for Krystexxa. The crux of the CRL issues were related to the drug’s API manufacturing process, not its fill/finish. The API supplier is BTG.

Chronic gout that is refractory to conventional therapy occurs in patients who have failed to normalize serum uric acid and whose signs and symptoms are inadequately controlled with xanthine oxidase inhibitors at the maximum medically appropriate dose or for whom these drugs are contraindicated.