STOCKHOLM, November 29, 2016 /PRNewswire/ --
Pharmalink AB, a specialty pharma company, announces that the European Commission (EC) has granted Orphan Medicinal Product (OMP) designation in the European Union (EU) to Nefecon® (budesonide), the Company's treatment for patients with primary IgA nephropathy (IgAN).
Nefecon, which already has orphan designation for IgAN in the US, has successfully completed a randomized, placebo-controlled Phase 2 study (the NEFIGAN trial) in more than 150 IgAN patients at risk of developing end stage renal disease (ESRD), under standardized rigorous blood pressure control. Full data and analysis of the trial is expected to be published in a peer-review journal. A Phase 3 registration trial is currently being planned.
Johan Häggblad, Pharmalink CEO, commented: "Receiving orphan drug designation from the EC, in addition to the previous orphan drug designation granted by the US FDA, is an important event for Pharmalink. It highlights the need for new products for the treatment of primary IgAN as well as our commitment to developing Nefecon for these patients."
Orphan drug designation is granted to drugs that are intended for the treatment of life threatening or chronically debilitating rare diseases where no therapeutic options either exist or are satisfactory. Rare diseases are those defined as having a prevalence of less than five in 10,000 in the European Union. The designation provides sponsors with development and commercial incentives, including 10 years of market exclusivity, prioritized consultation by the European Medicines Agency (EMA) on the development of the drug, including clinical studies, and certain exemptions from or reductions in regulatory fees. Click here for more information.
Notes to Editors
About IgA Nephropathy
IgA nephropathy (IgAN) is the most common form of glomerulonephritis (inflammation of the kidney glomeruli). The disease is characterized by deposits, predominantly containing polymeric IgA antibody, in the kidney that cause inflammation and renal damage.
IgAN can occur at any age, but the clinical onset is commonly during the second or third decades of life. It has been estimated that up to 40% of patients with IgAN progress to renal failure, often referred to as end-stage renal disease (ESRD) within 5-30 years following diagnosis. This patient population is estimated to at least 200,000 in major markets.
Patients suffering renal failure require dialysis or kidney transplantation. IgAN accounts for 10% of renal transplants among patients with primary glomerulonephritis in the US and between 7-20% of patients in Europe and Australia in long-term dialysis and renal transplantation programs.
Nefecon is a treatment for patients with primary IgA nephropathy (IgAN) at risk of developing end-stage renal disease (ESRD). Nefecon has successfully completed a randomized, placebo-controlled Phase 2b study in more than 150 IgAN patients at risk of developing ESRD, under standardized rigorous blood pressure control with an angiotensin-converting enzyme inhibitor (ACEI) and/or angiotensin II receptor I blocker (ARB). Full data and analysis of the NEFIGAN trial is expected to be published in a peer-review journal. A Phase 3 registration trial is currently being planned.
Nefecon is an oral formulation of a locally-acting and potent corticosteroid, budesonide, that down-regulates the disease process in the kidney through suppression of the gastrointestinal immune system thus exploiting the pivotal role the gastrointestinal tract plays in the overall immune response. Promising results indicate that treatment with Nefecon may provide clinical benefits to IgAN patients at risk of progressing to ESRD, and provide an alternative to dialysis and transplantation. Nefecon has received orphan drug designation by the US Food and Drug Administration (FDA).
About Pharmalink AB
Pharmalink is a specialty pharma company developing high value products for patients with significant unmet medical needs. With a highly experienced, dynamic management team, Pharmalink draws on its extensive experience of pharmaceutical development and marketing to efficiently identify and progress valuable and de-risked products.
Visit http://www.pharmalink.se for further information.
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SOURCE Pharmalink AB