Grifols has received US FDA approval for an expanded indication of Thrombate (antithrombin III [human]) to include pediatric patients with hereditary antithrombin deficiency (hATd). With this approval, Thrombate becomes the only antithrombin concentrate approved for both adults and children with this rare blood clotting disorder, addressing a critical gap in treatment options for pediatric patients.

Hereditary antithrombin deficiency is associated with a high risk of thrombotic events, with studies showing that up to 85% of patients experience at least one clotting episode by age 50. The FDA approval was based on data extrapolated from two clinical trials in adults, reflecting decades of successful use of antithrombin concentrate in treating adult patients.

“This label expansion helps close a long-standing gap in the treatment of pediatric patients with hereditary antithrombin deficiency,” said George M. Rodgers, professor of medicine in the Division of Hematology and Hematologic Malignancies at the University of Utah School of Medicine. “It gives clinicians added confidence that ATc can be appropriately used in children.”

Roland Wandeler, president of Grifols Biopharma, said the expanded indication provides a new therapeutic option for children and families living with hATd. “Expanding the indication to include pediatric patients provides a new option for children and families facing hereditary antithrombin deficiency,” Wandeler said. “This is another example of Grifols’ continuing work to provide more patients with medicines to enhance their health and well-being.”

Treatment of thromboembolism in patients with hATd typically involves anticoagulation therapy, sometimes complemented by antithrombin replacement using Thrombate or fresh frozen plasma in high-risk situations. Thrombate has been available in the U.S. for adult patients with hATd since 1991 and is indicated for treatment and prevention of thromboembolism, including perioperative and peripartum periods.

The FDA’s expanded label confirms that Thrombate can be safely and effectively used in children, providing clinicians with a well-characterized therapeutic option for a rare and high-risk patient population.