Savient Pharmaceuticals, Inc. announced that the US Food and Drug Administration (FDA) has approved Krystexxa (pegloticase), a PEGylated uric acid specific enzyme indicated for the treatment of chronic gout in adult patients refractory to conventional therapy.
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. Krystexxa is not recommended for the treatment of asymptomatic hyperuricemia.
"Krystexxa is the first-ever and only treatment approved by the FDA for adult patients who suffer with chronic gout that is refractory to conventional therapy," said Paul Hamelin R.Ph., president of Savient Pharmaceuticals. "The clinical data have demonstrated that many patients treated with Krystexxa 8 mg administered every two weeks can experience within six months of treatment significant positive clinical improvement reversing the course of this severe, crippling and debilitating disease. A statistically significant proportion of patients in our pivotal clinical trials achieved a lowering of their serum uric acid level to a mean of 0.7 mg/dL and achieved a complete response for the resolution of tophi within the first six months of therapy. We believe that the approval of Krystexxa is a significant step towards realizing our mission of transforming the lives of the patients in the US suffering with chronic gout refractory to conventional therapy, as many of them finally have a treatment that gives them hope of reversing this severely debilitating disease."
Savient expects Krystexxa to be available by prescription in the US later this year and believes it is well advanced in its preparations for the US launch of Krystexxa. Specific timing for the launch of Krystexxa will be determined within the context of the company's commercialization plan and by the progress and status of the company's efforts to pursue a strategic transaction for the sale of Savient. Krystexxa was granted an Orphan Drug designation by the FDA in 2001 that the company expects will provide the drug seven years of orphan drug market exclusivity. The composition, manufacture and methods of use and administration of Krystexxa are also the subject of a broad portfolio of patents and patent applications that the company expects will provide protection into 2026.
Savient will conduct a post-approval observational safety study in 500 patients treated for one year to further evaluate the frequency and severity of infusion reactions, anaphylaxis and immune complex-related adverse events, and to identify serious adverse events associated with Krystexxa therapy.
Savient has worked with the FDA to create a Risk Evaluation and Mitigation Strategy (REMS) programme to help physicians, healthcare providers and patients make treatment decisions for adults who suffer with chronic gout that is refractory to conventional therapy based on the Krystexxa comprehensive and current benefit:risk information. The Krystexxa REMS programme consists of a communication plan for health care providers and a medication guide for patients.
Krystexxa (pegloticase) is a PEGylated uric acid specific enzyme indicated for the treatment of chronic gout in adult patients refractory to conventional therapy. Krystexxa is not recommended for the treatment of asymptomatic hyperuricemia. Savient has exclusively licensed worldwide rights to the technology related to Krystexxa and its uses from Duke University (Duke) and Mountain View Pharmaceuticals, Inc. (MVP). Duke developed the recombinant uricase enzyme and MVP developed the PEGylation technology used in the manufacture of Krystexxa. MVP and Duke have been granted US and foreign patents disclosing and claiming the licensed technology and, in addition, Savient owns or co-owns US and foreign patents and patent applications, which collectively form a broad portfolio of patents covering the composition, manufacture and methods of use and administration of Krystexxa.
Savient Pharmaceuticals, Inc. is a specialty biopharmaceutical company focused on developing Krystexxa (pegloticase) for the treatment of chronic gout in patients refractory to conventional therapy.