Swedish firm Dilaforette has received orphan drug designation from the US Food and Drug Administration (FDA) for its sevuparin (DF02) to treat patients with sickle-cell disease (SCD).

With an aim to start recruitment of patients during the first half of 2015, the firm is currently in the final stage of study preparation for a Phase II study in SCD with sevuparin.

Sevuparin is a proprietary polysaccharide drug that can restore blood flow and prevent further microvascular obstructions caused by abnormal blood cells in SCD patients.

"Continued interactions with FDA and regional expert clinicians will enable future clinical development of sevuparin in the US."

Dilaforette CEO Christina Herder said: "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 US."

According to the company, the drug has anti-adhesive properties that could treat underlying cause of vaso-occlusive crisis (VOC) in SCD patients, with earlier pain relief, shorter hospital stay and reduced need of opioids.

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In January this year, the company obtained positive opinion from the Committee for Orphan Medicinal Products (COMP) on orphan drug designation in the EU for sevuparin to treat SCD. The opinion will be reviewed by the European Commission for final ratification.

Image: Very high-magnification micrograph of cirrhosis due to sickle cell disease. Photo: courtesy of Nephron.