Affymax Inc.,a biopharmaceutical company, and Takeda Pharmaceutical Company Limited, a research-based global company, have received the US Food and Drug Administration (FDA) approval for Omontys (peginesatide) injection for the treatment of anaemia due to chronic kidney disease (CKD) in adult patients on dialysis.

Omontys is the only once-monthly erythropoiesis-stimulating agent (ESA) for anemia to be made available to the dialysis patient population in the United States.

Omontys (peginesatide) injection is a synthetic, pegylated, peptide-based ESA. It is the only ESA that is peptide-based and its building blocks (amino acids) are arranged in a different order than erythropoietin (i.e., it has no sequence homology to endogenous erythropoietin). It was discovered by Affymax and will be co-commercialized in the United States by Affymax and Takeda. In February 2012, Takeda and its wholly-owned subsidiary, Takeda Global Research & Development Centre (Europe) Ltd., announced the acceptance of a Marketing Authorization Application for peginesatide by the European Medicines Agency. The application is currently under review by that agency.

The FDA's decision was based on a New Drug Application (NDA), which included results from two randomized, controlled, open-label, phase III studies (EMERALD 1 and 2) that demonstrated the safety and efficacy of Omontys dosed once monthly, compared to epoetin dosed between one-to-three times per week (according to product labels), in maintaining hemoglobin (Hb) levels in anemic CKD patients on dialysis. In these studies, the most commonly reported adverse reactions were shortness of breath, diarrhea, nausea, cough and arteriovenous fistula site complication.

The EMERALD studies were part of the largest clinical programme to support the NDA of an ESA in the treatment of anaemia in CKD. Enrolling 2,606 patients, including approximately 1,600 dialysis patients, the Omontys phase III programme was also the first to prospectively compare, in a head-to-head manner, the cardiovascular safety of different ESAs. Cardiovascular safety was evaluated based on a composite cardiovascular safety endpoint adjudicated by a blinded and independent committee.

In the approval action letter, the FDA outlined post-marketing requirements: an observational study and a randomized controlled trial to be completed with final reports submitted in 2018 and 2019, respectively. The objectives of the studies are to evaluate cardiovascular safety and assess safety of long-term use in adult patients on dialysis, in particular in the incident patient population. In addition, the post-marketing commitment includes the initiation of pediatric studies with target dates for completion between 2016 and 2027. Letters will be sent to nephrology healthcare providers as part of a Risk Evaluation and Mitigation Strategy (REMS) to inform them that Omontys is not indicated in patients with CKD not on dialysis. In two trials of Omontys, patients with CKD not on dialysis experienced increased specific cardiovascular events.

“The approval of Omontys now provides a therapeutic alternative to treat anemia of CKD in adult patients on dialysis, one of the most common complications affecting this patient population,” said John Orwin, chief executive officer, Affymax. “For over two decades, doctors have relied primarily on one erythropoietin-based treatment in the dialysis setting. With Omontys, doctors and patients will have access to a once-monthly alternative for the treatment of anemia in adult CKD patients on dialysis.”

“The FDA's approval of Omontys signifies an important milestone for the partnership between Takeda and Affymax as we fulfill our goal of providing an important new treatment option for the hundreds of thousands of CKD patients on dialysis who live with anaemia,” said Azmi Nabulsi, MD, president, Takeda Global Research & Development Centre, Inc. “Omontys is an example of our commitment to making treatment options available that accommodate the needs of evolving healthcare markets, such as the renal community.”

Anaemia (a condition in which blood has a lower than normal number of red blood cells) is a common complication in dialysis patients because their kidneys no longer produce enough erythropoietin, the hormone that stimulates red blood cell production in the body. According to the centres for Medicaid and Medicare Services (CMS), nearly 95 per cent of dialysis patients in the United States are being treated for anaemia with ESAs. Until the approval of Omontys, ESAs were recombinant (genetically engineered) versions of endogenous erythropoietin (erythropoietin that is made in the patient's body) that are injected up to three times a week. Omontys is a synthetic, pegylated, peptide-based ESA that is dosed once monthly.

“For dialysis patients, anaemia is another aspect of their challenging condition that must be addressed,” said Brigitte Schiller, MD, chief medical officer, Satellite Healthcare, Inc. “As a nephrologist who oversees the care of adult CKD patients on dialysis, I am glad to now have another option for the treatment of anaemia.”