Savient updates on its Biologics License Application with the FDA for KRYSTEXXA

Sep 16 2009

Savient Pharmaceuticals, Inc. (Nasdaq: SVNT) announced that on September 14, 2009, the Company completed a Type A meeting with the U.S. Food and Drug Administration (FDA) to discuss the Complete Response Letter (CRL) received by Savient on July 31, 2009 regarding the Company's Biologics License Application (BLA) for KRYSTEXXA(TM) (pegloticase) as a treatment for chronic gout in patients refractory to conventional therapy.

Based on the results of this meeting with the FDA, Savient believes that the FDA supports its approach to resolve all issues cited in the CRL. The meeting also confirmed that the FDA does not expect further clinical trials to be required as a result of Savient's reversion to the original manufacturing process to produce the Phase 3 clinical trial material for KRYSTEXXA, provided that no significant differences are observed between the material produced with the validated Phase 3 process and the Phase 3 clinical trial material. Savient, therefore, continues to believe that it can meet its previously discussed timeline of filing the resubmission in response to the CRL in early 2010.

"We are encouraged by the FDA's continuing collegial and collaborative dialogue in providing guidance to assist us in establishing the path forward to our resubmission for KRYSTEXXA," stated Paul Hamelin, President of Savient Pharmaceuticals. "We are grateful to the FDA for granting us this meeting so quickly and appreciate the considerable time and effort devoted by the FDA reviewers in evaluating our pre-meeting package and in providing very valuable feedback and guidance."

In response to the CRL, Savient requested and was granted the Type A meeting with the FDA to discuss, clarify and reach alignment on a resubmission plan to fully address all deficiencies and issues identified in the CRL. The FDA indicated that, in its view, Savient's plan to revert to and validate the original manufacturing process used to produce the Phase 3 clinical trials material, together with the inclusion of additional 0.22 micron filters in the manufacturing process, is a reasonable approach that the FDA expects will produce drug substance that is representative of that used in the pivotal Phase 3 clinical trials. The FDA stated that it also expects that the comparability between material produced with the validated Phase 3 process and the Phase 3 clinical trial material used in the replicate clinical trials to establish safety and efficacy can be sufficiently established by quality criteria alone without the need to conduct additional clinical studies, provided no significant differences between products are observed.

The FDA also agreed at this meeting with the methods and criteria proposed by Savient to tighten manufacturing analytical methods and acceptance criteria for all manufacturing steps in the final commercial production process. However, some of these final analytical methods and acceptance criteria are subject to being set based upon the data from historical manufacturing and validation batches that will be included in the resubmission. The meeting outcomes confirm Savient's belief that it is on track to meet its previously discussed timeline of filing the resubmission in response to the CRL in early 2010.

The FDA also provided additional clarity relating to the steps necessary for Savient's drug substance manufacturer, Bio-Technology General (Israel) Ltd (BTG), a subsidiary of Ferring Pharmaceuticals, to satisfactorily correct the observations cited by the FDA during its pre-approval inspection of the BTG manufacturing facility. Savient believes that BTG's remediation of these observations can be corrected ahead of its resubmission to the FDA in response to the CRL.

The FDA also stated that the format and content of Savient's proposed safety update, which will include additional data collected from the KRYSTEXXA Open Label Extension study, is acceptable to the FDA.

During the meeting, the Company was informed that the review cycle for the resubmission would include the review of all data to fully address all issues identified in the CRL, including the final product labeling and the REMS materials. Since the resubmission will include REMS materials, this is subject to a Class 2 review cycle, meaning simultaneous approval of all components of our filing within six months of the date of our resubmission.

"We look forward to continuing to work with the FDA and executing on our resubmission strategy to the Complete Response Letter so that we can move forward with delivering this important therapy to treat chronic gout patients who are suffering from this crippling, debilitating disease and who have no other treatment options," commented Mr. Hamelin.