Vifor Pharma announced that the first patient has been enrolled in a double-blind, randomized phase-IIa clinical trial evaluating the safety, efficacy and tolerability of vamifeport in adult patients with sickle cell disease. Vamifeport, developed by Vifor Pharma, is a novel oral ferroportin inhibitor investigated for treatment of diseases characterized by ineffective production of red blood cells and iron overload, including SCD and beta-thalassemia. It has been granted orphan drug designation from both the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) for the treatment of SCD.
Dr. Klaus Henning Jensen, Chief Medical Officer of Vifor Pharma Group commented, “Starting the phase-IIa trial is an important milestone in the clinical development of vamifeport for sickle cell disease, a debilitating orphan condition for which there are very limited treatment options. Patients worldwide continue to suffer from consequences of the disease which dramatically impacts their quality of life and life expectancy. We look forward to continuing development of our potential new treatment for these patients”.
SCD is a group of inherited red blood cell disorders and is a genetic condition present at birth. In this disease, red blood cells carry abnormal haemoglobin, which makes them prone to rupture, causing adhesion of sickle cells and inflammatory cells to the blood vessels. This ultimately leads to an obstruction of blood flow and organ damage. There are an estimated 150,000 patients in Europe and the US combined living with sickle cell disease.
The phase-IIa study is a randomized, double-blind, placebo-controlled, parallel group trial aiming to recruit 25 patients at 14 clinical sites in the United States, the United Kingdom, Lebanon and Greece. The primary objective of the trial is to assess the change in markers of hemolysis (the rupturing of red blood cells) upon treatment with vamifeport. Patients will undergo initial treatment for 4 weeks, followed by an additional 4-week period with either maintenance of the dose level or a dose step-up. The study also includes a 4-week follow-up period after treatment. End of enrolment is expected mid-2022 with topline results anticipated at the end of 2022.
