Dilaforette AB has announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation for sevuparin (DF02) for the treatment of patients with sickle-cell disease (SCD). The company is currently in the 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 the 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 the final stage of preparation for a Phase II study and aim to start recruitment of patients during the first half of 2015.
"An Orphan Drug Designation in the US is an important step in our efforts to bring an important new, valuable and needed treatment to SCD patients. The designation gives advantages in FDA assistance, user-fee benefits and, after orphan drug registration, seven years of market exclusivity. Continued interactions with FDA and regional expert clinicians will enable future clinical development of sevuparin in the U.S.," 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. In the US, it estimated that close to 100,000 patients are diagnosed with this hereditary disease.
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.