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Hematology
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FDA grants orphan drug designation to sickle cell disease treatment

Posted on November 3, 2020

The U.S. Food and Drug Administration has grant orphan drug designation to investigational, plasma-derived hemopexin therapy (CSL889; CSL Behring) for the treatment of sickle cell disease (SCD).

“Having treated hundreds of adults and children living with sickle cell disease over 30 years, I’m intensely aware of the need for novel and effective therapies, especially to relieve the tremendous pain from VOC,” said Professor Greg Kato, who is leading the clinical development of CSL 889 at CSL Behring in a press release. “This newly granted orphan status recognizes the urgency for progressing new treatment options into the clinic.”

Read the full press release here.

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