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Swedish Pharmalink receives Orphan Drug Approval for Busulipo

Authored by James Sheppard

The Swedish speciality pharmaceutical company Pharmalink AB announced that it had received Orphan Drug approval from the US FDA for oncology conditioning agent Busulipo. Busulipo is indicated for use in cancer patients prior to hematopoietic stem cell transplantation (HSCT).

Pharmalink AB has been developing Busulipo as a liposome/lipid complex formulation that improves the safety and stability of the chemotherapy agent busulfan. The concept behind Busulipo was originally developed at Karolinska Institute (Sweden) and was acquired by Pharmalink AB in 2006.  Pharmalink AB is intending for Busulipo to become the new ‘gold standard’ in conditioning prior to HSCT. An early clinical trial, involving 90 patients has showed that Busulipo is safe and effective. The clinical study provided extensive data on efficacy, safety and pharmacokinetics and this data compares positively with historical controls.

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.’

The news follows soon after Pharmalink AB announced it has signed a manufacturing agreement with Spanish biotechnology company GP Pharm for the manufacture of Busulipo. Further to this, Pharmalink AB has recently completed a successful series C funding round raising $15m for the development of Busulipo and to complete a phase IIb study of Nefecon. Nefecon is a potentially disease modifying treatment indicated in patients with primary IgA nephropathy at risk of developing end-stage renal disease. The funding round was led by a new investor, Investinor. Investinor is the Nordic areas largest venture capital group.


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