Pharmalink AB's product, Busulipo, has received orphan drug designation from the US Food and Drug Administration (FDA). The Company is currently preparing for registration trials with an optimized 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 Haggblad, 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....