Akebia自研药物vafseo获欧盟委员会批准，治疗成人慢性肾脏疾病透析相关症状性贫血

On April 26, Akebia Therapeutics announced that the European Commission (EC) had approved the marketing of its self-developed drug vafseo (vadustat). Vafseo is an oral hypoxia-inducible factor prolyl hydroxylase (HIF-PH) inhibitor indicated for the treatment of symptomatic anemia associated with chronic kidney disease (CKD) in adults on long-term maintenance dialysis. Currently, vafseo is approved in 32 countries, and this approval makes the drug available in all 27 EU member states.

Anemia is prevalent among hemodialysis patients and may be associated with a number of adverse clinical outcomes. Across Europe, more than 200,000 dialysis patients are currently being treated for CKD-related anemia. Insufficient secretion of erythropoietin. It is the main cause of renal anemia. The lifespan of red blood cells is shortened. The accumulation of toxins in the body will shorten the life of red blood cells, and uremic toxins and red blood cell inhibitory factors affect bone marrow hematopoiesis. Patient malnutrition can lead to iron deficiency anemia, which is mainly related to insufficient iron intake, gastrointestinal bleeding and hemodialysis losses. Hemodialysis patients may also suffer from hidden bleeding in the digestive tract, bleeding from internal fistulas, residual blood from the dialyzer, etc., resulting in chronic blood loss.

Vafseo is an oral hypoxia-inducible factor prolyl hydroxylase inhibitor (HIF-PHI) designed to mimic the body's physiological effects of hypoxia at high altitude. At high altitudes, the body responds to oxygen scarcity by increasing the production of hypoxia-inducible factor (HIF). HIF regulates the mobilization of iron and the production of erythropoietin (EPO) to stimulate the production of red blood cells, thereby improving oxygen transport. Research on the oxygen sensing signaling pathway won the Nobel Prize in Physiology or Medicine in 2019.

The approval comes after the European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP) issued a positive opinion in February 2023 recommending that the European Commission approve vafseo. Based on data from comprehensive development programs involving more than 7,500 patients, including the global Phase III clinical program evaluating vafseo for the treatment of anemia due to CKD in adult patients on dialysis (INNO2VATE).

INNO2VATE trial data showed that vafseo met the primary treatment endpoint and key secondary treatment endpoints in each of the two INNO2VATE studies, showing non-inferiority to alpha-dapoetin, measured as the mean change in hemoglobin (Hb) between baseline and the primary evaluation period (weeks 24 to 36) and the secondary evaluation period (weeks 40 to 52). Vafseo also met the primary safety endpoint of INNO2VATE, which was non-inferiority of vafseo to alpha-dapoetin in time to first major adverse cardiovascular event.

John P. Butler, CEO of Akebia, commented: "We are very pleased that the European Commission has approved vafseo, which is an important milestone for Akebia, but has an even greater impact on the hundreds of thousands of European patients diagnosed with anemia associated with CKD on dialysis. We believe patients on chronic maintenance dialysis will benefit from more treatment options. Following approval, we are eager to select a European partner who can quickly bring vafseo to these patients."

"We are grateful to the patients, physicians, investigators and site coordinators who participated in our clinical trials and whose efforts resulted in this important approval. I also want to express my deep gratitude to our team at Akebia, as this approval is the culmination of years of work and demonstrates their commitment to improving the lives of patients affected by kidney disease."