“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.
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 FDA’s Rare Pediatric Disease Priority Review Voucher program, a sponsor who receives an initial approval for a drug or biologic for a “rare pediatric disease” may qualify for a voucher that can be redeemed to receive a priority review of a subsequent marketing application for a different product.
SCD is the most common inherited blood disorder in the United States, impacting approximately 100,000 people. Worldwide, approximately 100 million people carry the SCD trait and an estimated five million live with the disease.
While the majority of people with SCD are of African descent, the disease can affect all ethnic groups, especially those from areas where malaria is or was endemic, such as the Middle East, India and the Southern Mediterranean. Acute pain crises or VOCs are the most common clinical manifestation of SCD. A VOC occurs when hypoxia and inflammation lead to vascular occlusion, tissue ischemia and pain.
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 June 2020 in advance of a poster shown at the Foundation for Sickle Cell Disease Research Meeting on September 24, 2020.
Source: Company Press Release