GlycoMimetics, Inc. announced that the U.S. Food and Drug Administration (FDA) has granted the Company a Rare Pediatric Disease designation for rivipansel for the treatment of sickle cell disease in patients 18 years old and younger. This designation recognizes the significant needs in pediatric patients.
“The FDA’s designation recognizes the morbidity and mortality burdens of sickle cell disease as well as its significant impact during childhood with life-long implications. With this designation, the agency acknowledges the urgent need for improved treatment of children living with sickle cell disease,” stated Helen Thackray, Chief Medical Officer of GlycoMimetics.
“With global rights for rivipansel in our hands, we are exploring options to move forward in sickle cell disease, including discussions with the FDA as to whether there is a regulatory path to approval,” added Rachel King, Chief Executive Officer. “We plan to roll out the full data set from the Phase 3 RESET program at upcoming medical meetings, based on the acceptance of abstract submissions.”