FDA grants orphan drug designation to CSL889 for sickle cell disease
Click Here to Manage Email Alerts
The FDA granted orphan drug designation to CSL889 for the treatment of sickle cell disease, according to the agent’s manufacturer.
CSL889 (CSL Behring) is an investigational plasma-derived hemopexin therapy.
Low hemopexin levels have been linked to increased symptoms — particularly vaso-occlusive crises — among people with sickle cell disease.
“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 vaso-occlusive crises” Gregory Kato, MD, senior director of global clinical programs for hematology at CSL Behring, said in a company-issued press release. “This newly granted orphan status recognizes the urgency for progressing new treatment options into the clinic.”
The FDA 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. The designation allows manufacturers to qualify for various incentives, including tax credits for qualified clinical trials and — upon regulatory approval — 7 years of market exclusivity.
Read more about
Click Here to Manage Email Alerts