Savient has received a complete response letter from FDA. The agency stated that it can not approve the company’s Biologics License Application (BLA) for KRYSTEXXA (pegloticase) at this time as a treatment for chronic gout in patients refractory to conventional therapy.
Reportedly, the complete response letter from FDA cites deficiencies with the chemistry, manufacturing and controls (CMC) section of the BLA, and also provided the current draft of the proposed labeling and further guidance regarding a Risk Evaluation and Mitigation Strategy (REMS).
FDA has concluded that the comparability data submitted for the material manufactured using the proposed commercial manufacturing process, was not adequate to demonstrate that it was representative of the material used to establish the safety and efficacy of KRYSTEXXA in its phase 3 clinical trials.
FDA stated that the company has the option of either reverting to and validating the manufacturing process used to produce KRYSTEXXA for the phase 3 clinical trials, or conducting additional comparability clinical trials to support the use of KRYSTEXXA manufactured using the proposed commercial manufacturing process.
Moreover, the company was also informed that its resubmission to the FDA, in response to the complete response letter, must include an update of safety data from all on-going studies.
Paul Hamelin, president of Savient, said: While our timeline for resubmission to the FDA is subject to a number of uncertainties, we currently believe that we can target completion of our resubmission for early 2010. We hope to have more clarity on the expected timeline after we meet with the FDA to discuss the complete response letter.
While we believe we have made substantial progress toward the potential final approval of KRYSTEXXA, we also have more work to do with the FDA to resolve these open issues. We are committed to work diligently to address these issues with a goal of obtaining final approval for KRYSTEXXA so we can provide this therapy to those chronic gout patients who are suffering from this crippling, debilitating disease and have no other treatment options, he added.