Having partnered last year with an international team that surveyed the genomes of 12, 000 individuals to find a genetic cause for gout, Johns Hopkins scientists now have shown that the malfunctioning gene they helped uncover can lead to high concentrations of blood urate that forms crystals in joint tissue, causing inflammation and pain the hallmark of this disease. The ABCG2 gene, they found, makes a protein that normally transports urate out of the kidney and into urine before the waste product does any harm. In studies using frog egg cells genetically engineered with human DNA, the Hopkins researchers established the role of the ABCG2 gene as a cause of gout, lending credence to suspicions that metabolic deficiencies, in addition to too much rich food and alcohol, are mostly to blame for this painful type of arthritis that affects 3 million Americans.
Ardea Biosciences Initiates Phase 2b Clinical Trial Of RDEA594, Lead Product Candidate For The Treatment Of Gout
Ardea Biosciences, Inc. (Nasdaq:RDEA) today announced that it has initiated a Phase 2b clinical trial of RDEA594, its lead product candidate in development for the management of hyperuricemia and gout. The Company also announced the selection of RDEA684, a next-generation URAT1 inhibitor, as a development candidate for the same indication. The randomized, double-blind, placebo-controlled, dose-response study will evaluate the safety and serum urate-lowering effects of 200, 400 and 600 mg of RDEA594 in a total of 140 gout patients with hyperuricemia (uric acid levels of 8 mg/dL or more). The primary efficacy endpoint is the proportion of patients whose serum urate level is less than 6.0 mg/dL following four weeks of treatment. This study will be conducted at multiple sites in Europe and North America, with initial results expected by the end of 2009.
The U.S. Food and Drug Administration has approved Colcrys to treat acute flairs in patients with gout, a recurrent and painful form of arthritis, and patients with familial Mediterranean fever (FMF), an inherited inflammatory disorder. The medication's active ingredient is colchicine, a complex compound derived from the dried seeds of a plant known as the autumn crocus or meadow saffron (Colchicum autumnale). Colchicine has been used by healthcare practitioners for many years to treat gout but had not been approved by the FDA. The FDA has an initiative underway to bring unapproved, marketed products like colchicine under its regulatory framework. This initiative promotes the goal of assuring that all marketed drugs meet modern standards for safety, effectiveness, quality and labeling.
Savient Pharmaceuticals Receives Complete Response Letter From U.S. Food And Drug Administration For KRYSTEXXA TM
Savient Pharmaceuticals, Inc. (Nasdaq: SVNT) announced that the Company has received a complete response letter from the U.S. Food and Drug Administration (FDA) stating that the FDA can not at this time approve the Company's Biologics License Application (BLA) for KRYSTEXXA(TM) (pegloticase) as a treatment for chronic gout in patients refractory to conventional therapy. The complete response letter from the 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) (Medication Guide and Communication Plan). The Company intends to immediately request a meeting with the FDA to discuss and clarify the issues raised in the complete response letter.
EnzymeRx, LLC a clinical-stage biotechnology company, announced that its Investigational New Drug (IND) application for Uricase-PEG 20, filed with the U.S. Food and Drug Administration (FDA) last month, has become effective. EnzymeRx filed this IND for intravenous Uricase-PEG 20 in the management of elevated uric acid levels associated with tumor lysis syndrome. Tumor lysis syndrome is a serious condition that can occur during the treatment of certain tumors, and can result in sharply elevated uric acid levels. Uricase-PEG 20 metabolizes poorly soluble uric acid into highly soluble allantoin, and in a prior phase 1 clinical trial, it was shown to lower uric acid levels in gout patients. Because its long half-life may provide for an extended duration of uric acid-lowering after just a single dose, Uricase-PEG 20 may offer a convenient alternative to existing therapies for tumor lysis syndrome.