Pegloticase, a recombinant formulation of porcine uricase, has been approved by the Food and Drug Administration for the treatment of chronic gout in adults who are refractory to conventional treatments, the agency announced on Sept. 14.
Pegloticase, which is administered intravenously every 2 weeks, is approved with a Risk Evaluation and Mitigation Strategy (REMS), which will include materials for health care practitioners and a patient medication guide that explain the risks of severe infusion and allergic reactions associated with treatment, the FDA said in its statement.
It will be marketed under the trade name Krystexxa, by Savient Pharmaceuticals Inc. About 3% of the 3 million adults who have gout do not benefit from conventional therapy, and pegloticase “offers an important new option for them,” Dr. Badrul Chowdhury, director of the division of pulmonary, allergy, and rheumatology products in the FDA’s Center for Drug Evaluation and Research, said in the FDA statement.
Uricase is an enzyme that lowers serum uric acid and is not produced by humans.
In two clinical trials of 212 patients with severe gout, treatment with pegloticase lowered uric acid levels and reduced deposits of uric acid crystals in joints and soft tissue, according to the FDA.
In June 2009, an FDA advisory panel recommended approval of the drug for treating refractory gout based on these studies, agreeing that it would meet an unmet medical need. At the meeting, several rheumatologists on the panel referred to the drug’s unique ability to reduce tophi in patients in clinical trials.
In the studies, one of every four patients had a severe allergic reaction to the infusion, so patients receiving an infusion of pegloticase should be pretreated with a corticosteroid and antihistamine to minimize the risks of allergic reactions. Because the drug was not studied in patients with heart failure, “physicians are also being warned to be cautious” about administering the drug to patients with heart failure, the statement said.
In a statement, Savient said that pegloticase would be available “later this year.”