Peginesatide, a long-acting erythropoiesis-stimulating agent, has been approved as a treatment for anemia caused by chronic kidney disease in adults who are on dialysis, the Food and Drug Administration announced March 27.
This is the first erythropoiesis-stimulating agent (ESA) approved for this indication since 2001, Dr. Richard Pazdur, director of the office of hematology and oncology products in the FDA’s Center for Drug Evaluation and Research, said in a statement. The product "offers patients and health care providers the convenience of receiving ESA therapy just once per month instead of more frequent injections," he noted.
The FDA approved the drug with a Risk Evaluation and Mitigation Strategy (REMS) that consists of educational elements for health care professionals and a requirement to assess data on how the drug is used, the statement said. The FDA can require a REMS for a product when the agency determines certain measures are needed to ensure that a drug’s benefits outweigh its risks.
Peginesatide is not approved for treating anemia in patients with chronic kidney disease (CKD) who are not on dialysis, for patients who are being treated for cancer and whose anemia is not caused by CKD, or for use instead of a red blood cell transfusion in patients who require immediate correction of anemia – which are approved indications for other ESAs. The label also states that treatment with peginesatide has not been shown to "improve symptoms, physical functioning, or health-related quality of life."
Peginesatide, which will be marketed as Omontys by Affymax Inc., is administered once a month, intravenously or subcutaneously. Treatment is started when the hemoglobin level is below 10 g/dL, according to the prescribing information.
Approval was based on two randomized open-label studies of 1,608 patients with CKD who were on dialysis, who, after their hemoglobin levels were stabilized with epoetin, were randomized to continue treatment with epoetin or peginesatide. Treatment with peginesatide was "as safe and effective as epoetin in maintaining hemoglobin levels within the studies’ prespecified range of 10 to 12 g/dL," according to the FDA statement. Diarrhea, vomiting, hypertension, and arthralgias were among the most common adverse effects reported in 10% or more of the patients on dialysis treated with peginesatide.
At a meeting in December 2011, the majority of an FDA advisory panel voted that peginesatide had a favorable risk-benefit profile for the approved indication. At that meeting, panelists were concerned about the higher rate of serious adverse events in patients treated with peginesatide, compared with those treated with darbepoetin, in studies of patients with anemia from CKD who were not on dialysis. But in the two studies of patients on dialysis, panelists agreed that the safety and efficacy of peginesatide were comparable with epoetin and cited the convenience of once-a-month dosing for patients.
Darbepoetin alfa, marketed as Aranesp, and epoetin alfa, marketed as Epogen and Procrit, are the ESAs approved in the United States; they are administered as often as one to three times per week. A pegylated epoetin beta (Mircera) is not available in the United States.
Like the other ESAs, the peginesatide prescribing information includes a boxed warning about the increased risks of deaths and serious cardiovascular events when higher hemoglobin levels are targeted, and the recommendation to use the lowest dose needed to reduce the need for transfusions.