Dilaforette AB has announced that it has received positive opinion from the Committee for Orphan Medicinal Products (COMP) on orphan drug designation in the EU for sevuparin (DF02) for the treatment of patients with sickle-cell disease (SCD).
The opinion will now be forwarded to the European Commission for final ratification. The company is currently in final stage of study preparation for a Phase II study in SCD with sevuparin and aim to start recruitment of patients during the first half of 2015.
Sevuparin is an innovative, proprietary polysaccharide drug, which has potential to restore blood flow and prevent further microvascular obstructions, caused by abnormal blood cells in SCD patients.
With its anti-adhesive properties, sevuparin could thereby offer treatment of the underlying cause of vaso-occlusive crisis (VOC) in SCD patients, with earlier pain relief, shorter hospital stay, reduced need of opioids and improved quality of life. Dilaforette is currently in final stage of study preparation for a Phase II study and aim to start recruitment of patients during the first half of 2015.
"An Orphan Drug Designation is a key step in our efforts to develop sevuparin for this severe and very debilitating disease. The designation will provide us with development and commercial incentives, including protocol assistance and scientific advice during product development, waiving or reduction of certain fees, eligibility for grants and R&D support initiatives as well as a 10-year market exclusivity” said Christina Herder, CEO of Dilaforette.
SCD is a disabling and potentially fatal disease with a large unmet medical need in both the developed and developing world. SCD patients undergo on average one VOC per year. This acute complication is caused by sickle blood cells obstructing the blood flow to organs leading to ischemia and often severe pain. Long-term, SCD patients are at risk of organ damage and premature death.