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FDA grants orphan drug designation to sevuparin for sickle cell disease
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The FDA granted orphan drug designation to sevuparin for the treatment of patients with sickle cell disease, according to a press release from the drug’s manufacturer.
Sevuparin (DF02, Dilaforette) is a proprietary polysaccharide drug which may restore blood flow and prevent further microvascular obstructions caused by abnormal blood cells in patients with sickle cell disease. The anti-adhesive properties of the agent could potentially treat underlying causes of vaso-occlusive crisis in patients with sickle cell disease, leading to earlier pain relief, shorter hospital stay, reduced need of opioids and improved quality of life, according to the release.
“An Orphan Drug Designation in the U.S. is an important step in our efforts to bring an important new, valuable and needed treatment to [sickle cell disease] patients,” Christina Herder, PhD, MBA, chief executive officer of Dilaforette, said in the press release. “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.”
Dilaforette plans to begin recruiting patients with sickle cell disease during the first half of 2015 for a phase 2 study evaluating sevuparin.
The FDA’s Office of Orphan Products Development grants orphan drug designation to novel drugs and biologics that are intended for the safe and effective treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the United States.