CSL889 Receives Orphan Drug Designation for Sickle Cell Disease

The FDA and the European Commission have granted orphan drug designation to CSL889, a plasma-derived hemopexin therapy, for the treatment of sickle cell disease (SCD).

In patients with SCD, low levels of hemopexin have been linked to increased incidence of acute vaso-occlusive crises (VOC), for which there is no approved treatment.

“Having treated hundreds of adults and children living with SCD over 30 years, I’m intensely aware of the need for novel and effective therapies, especially to relieve the tremendous pain from VOC,” Greg Kato, MD, Senior Director of Global Clinical Programs for Hematology at CSL Behring, said in a press release. “This newly granted orphan status recognizes the urgency for progressing new treatment options into the clinic.”

A phase I clinical trial of CSL889 hemopexin therapy for the treatment of VOC is ongoing.

Sources: CSL Behring press release, November 2, 2020; ClinicalTrials.gov, NCT04285827.