Horizon Pharma has done a retrospective analysis of data from previous pivotal, randomized KRYSTEXXA (pegloticase) clinical trials, which demonstrate that refractory chronic gout patients defined as “non-responders” based on serum uric acid levels in the trials still achieved significant clinical benefit despite a loss of response in uric acid levels.
These data (abstract 212) were presented during the American College of Rheumatology (ACR) and the Association of Rheumatology Health Professionals (ARHP) Annual Meeting in Washington D.C. on Sunday, November 13, 2016.
“In a recent survey of U.S. adults living with gout, nearly half said they could not imagine anything more painful than a gout flare, revealing a critical need for education and ongoing study of the available options for managing the condition,” said Jeffrey W. Sherman, M.D., FACP, executive vice president, research and development and chief medical officer, Horizon Pharma plc. “Our clinical presence at the ACR meeting this year is the largest for KRYSTEXXA in three years and underscores our drive to expand awareness of this innovative treatment option for refractory chronic gout sufferers.”
Horizon noted that KRYSTEXXA is the first and only medicine approved by the U.S. Food and Drug Administration for the treatment of refractory chronic gout, which represents an orphan disease subset of the common form of gout. In general, gout is a type of chronic inflammatory arthritis where uric acid builds up in the blood and can lead to severe pain and joint destruction, as well as the manifestation of tophi, which are hard, uric acid deposits under the skin that contribute to bone and cartilage destruction. Patients with refractory chronic gout continue to have abnormally high levels of uric acid despite conventional therapies. Refractory chronic gout impacts an estimated 50,000 people in the United States.
“The pivotal clinical trials evaluating KRYSTEXXA provide a wealth of data for health care providers treating people living with refractory chronic gout,” said Peter E. Lipsky, M.D., AMPEL BioSolutions, and one of the authors of the poster presentation. “A retrospective look at this data uncovered that patients defined as “non-responders” in the trial actually experienced significant clinical benefits from KRYSTEXXA, with a quarter of non-responders achieving complete tophus resolution at six months.”
Last week, Hhorizon published its quarterly results in which it was noted that orphan drugs, among which is Krystexxa, made one third of Horizon’s quarterly sales. On a GAAP basis in the third quarter of 2016, net loss was $5.9 million and non-GAAP adjusted net income was $115.5 million.