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Dilaforette gets FDA orphan drug status for sevuparin to treat sickle-cell disease

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

Currently, the company is in the final stage of study preparation for a Phase II trial in SCD with sevuparin and intends to begin patient enrollment during the first half of 2015.

Sevuparin is claimed to have the potential to restore blood flow and prevent further microvascular obstructions, caused by abnormal blood cells in SCD patients.

The company said that with its anti-adhesive properties, sevuparin could provide treatment of the underlying cause of vaso-occlusive crisis (VOC) in SCD patients.

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."

Sickle-cell disease is a disabling and potentially fatal disease with a large unmet medical need in both the developed and developing world.