Pharmalink AB, a privately held Swedish pharmaceutical company, has received orphan drug designation by the US Food and Drug Administration (FDA) for its product candidate Nefecon (PL-56) for patients with IgA nephropathy, also referred to as Berger´s Disease. This program is presently in clinical development phase II.
Johan Häggblad, Managing director of Pharmalink AB said:
“Today’s news marks a significant milestone for Pharmalink, and increases the commercial value of Nefecon® as it moves towards the market place. We believe this product candidate has great potential in treating IgA nephropathy. The orphan drug designation granted by the US FDA is very encouraging for our team as we now look to start executing on activities leading up to registration trials.”
Pharmalink AB previously announced global patent coverage and highly promising results from a planned interim analysis of an ongoing open Phase II trial which was designed to evaluate the efficacy and safety of Nefecon® in IgA nephropathy, a glomerulonephritis leading to end-stage renal disease (ESRD). The interim results were confirmed in the final results obtained in early 2009. Nefecon® is an oral small molecule product which downregulates the inflammatory process in the kidneys. It is being co-developed by Pharmalink and Archimedes Pharma under a partnership agreed in January 2004 to bring the product candidate to proof-of-concept. Orphan drug designation is intended to encourage companies to develop therapies for the treatment of diseases that affect fewer than 200,000 people in the US. It provides companies with market exclusivity and can also lead to certain financial incentives that can help support the development of a specific compound.
The concept underlying the renal disease product candidate Nefecon is a patented invention by Professors Bengt Fellström and Roger Hällgren at Uppsala University, Sweden. It is delivered using Archimedes’ proprietary TARGIT® drug delivery technology which enables the localized delivery of drugs to the lower small intestine or colonic regions of the gut. Archimedes is providing access to the TARGIT platform and providing all trial supplies of Nefecon. Pharmalink is responsible for the management of the clinical trials.