The US Food and Drug Administration (FDA) has approved an expanded indication for Grifols’ Thrombate III, allowing its use in pediatric patients diagnosed with hereditary antithrombin deficiency (hATd).
The decision marks the first time an antithrombin concentrate (ATc) has been cleared for both adult and pediatric populations with the condition, significantly broadening treatment access for affected families. The company described the milestone as a crucial advancement for a rare and frequently underdiagnosed blood-clotting disorder that may affect up to 700,000 people in the US.
Hereditary antithrombin deficiency is associated with a significantly elevated risk of abnormal blood clots. Research indicates that up to 85% of individuals with the disorder will experience at least one thrombotic event by age 50, underscoring the importance of early and effective therapeutic options.
Clinicians have long faced a gap in treatment guidelines for children with the condition. According to George M. Rodgers, III, M.D., Ph.D., of the University of Utah School of Medicine, the expanded indication “helps close a long-standing gap in the treatment of pediatric patients,” offering greater confidence in the appropriate use of ATc in younger patients.
FDA Approval
The FDA’s decision relied on data extrapolated from two clinical trials involving adult patients. These studies, which documented decades of safe and effective use, supported the conclusion that Thrombate III could also be administered appropriately in pediatric cases.
Roland Wandeler, president of Grifols Biopharma, highlighted the importance of the approval for families managing hATd. He noted that including pediatric patients “provides a new option for children and families” and reflects the company’s continuing commitment to expanding treatment access.
The updated label indicates Thrombate III for treatment and prevention of thromboembolism in both adult and pediatric patients, including perioperative and peripartum settings. Thrombate III, like other plasma-derived products, carries risks of hypersensitivity reactions and infectious agent transmission.
Grifol’s Pipeline
Last month, the company started the US commercial launch of its intravenous immunoglobulin (IVIg) therapy, Yimmugo. The US FDA approved the therapy for the treatment of primary immunodeficiencies (PID) in June 2024.
Grifols also expanded its US diagnostics manufacturing capacity with the launch of a new 73,541-square-foot facility in San Diego, California. The site will produce DG Gel cards and reagent red blood cells (RRBCs) technologies critical for ensuring blood transfusion compatibility and identifying clinical complications such as Rh incompatibility in pregnancy.
A late-stage therapy in the company’s pipeline is its fibrinogen concentrate therapy, BT524. In June, the company noted that therapy is on track for commercial rollout in Europe later this year, with US approval expected by early 2026.
