Pharmalink's Busulipo receives FDA orphan drug designation

Pharmalink AB, a specialty pharma company focused on orphan and niche products, today announces its product Busulipo™, a best-in-class conditioning agent for use in cancer patients prior to hematopoietic stem cell transplantation (HSCT; also known as bone marrow transplantation), has received orphan drug designation from the US Food and Drug Administration (FDA). The Company is currently preparing for registration trials with an optimised formulation of Busulipo as part of its plans to advance the product towards the market.

Busulipo™ has been developed by Pharmalink as a liposome/lipid complex formulation that improves the safety and stability of the chemotherapy agent busulfan, and is aimed at becoming the new gold standard for use in conditioning prior to HSCT. An early Busulipo formulation has successfully undergone clinical trials with more than 90 patients treated.

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 product.

Johan Häggblad, Managing Director of Pharmalink said: "The receipt of orphan drug designation for Busulipo is welcome news as we prepare for registration trials and advance the product towards market approval. We believe that the medical benefits of Busulipo have the potential to make it the drug product of choice in conditioning prior to HSCT, an important procedure that 18,000 American patients underwent in 2010. These numbers are expected to increase in the years to come as more patients become eligible for HSCT. Following our recent SEK96m financing, Pharmalink is well positioned to advance both Busulipo and our other lead product candidate, Nefecon®, through pivotal studies and towards commercialisation."

Nefecon is a disease-modifying medication with orphan drug designation and the first specifically aimed at treating patients with primary IgA nephropathy at risk of developing end-stage renal disease. Pharmalink is undertaking two pivotal clinical trials of Nefecon, which would be the basis for a market authorisation application. The first of these studies, a Phase IIb multi-center, randomized, double-blinded placebo-controlled study, began in December 2012. Results are expected in 2015 and will support a pivotal Phase III trial.