FDA Grants GlycoMimetics Rare Pediatric Disease Designation for Rivipansel for Treatment of Sickle Cell Disease
“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
“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
About Rare Pediatric Disease Designation
The FDA defines a "rare pediatric disease" as a serious or life-threatening rare disease in which the serious or life-threatening manifestations primarily affect individuals aged from birth to 18 years. Under the
About Sickle Cell Disease (SCD) and VOC
SCD is the most common inherited blood disorder in
About Rivipansel
Rivipansel, a glycomimetic drug candidate that binds to all three members of the selectin family (E-, P- and L-selectin), was GlycoMimetics’ first drug candidate to enter clinical development. After the Phase 3 RESET trial conducted by Pfizer, GlycoMimetics’ former collaborator, produced disappointing results in 2019, new clinical outcome data from a post hoc analysis of early treatment with rivipansel were published in
About
Forward-Looking Statements
This press release contains forward-looking statements regarding the clinical development and potential benefits and impact of the Company’s drug candidates. These forward-looking statements include those relating to the planned or potential clinical development of the Company’s product candidates, including the presentation of data from clinical trials. Actual results may differ materially from those described in these forward-looking statements. For a further description of the risks associated with these statements, as well as other risks facing
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