FDA Expands Sanofi’s Cablivi Approval to Pediatric Patients who have Acquired TTP
On Jan. 5, 2026, the US Food and Drug Administration (FDA) approved Sanofi’s Cablivi® (caplacizumab-yhdp) for injection to treat pediatric patients 12 years and older who have acquired thrombotic thrombocytopenic purpura (aTTP) in combination with plasma exchange and immunosuppressive therapy. Cablivi, originally approved in 2019 for adults, is a nanobody-based therapy that targets von Willebrand factor to prevent platelet aggregation and microvascular thrombosis. aTTP is a rare disorder causing small blood vessel clots that can block blood flow to vital organs, while platelet clumping and red blood cell destruction lead to low platelet counts and anemia. Approval for this population was supported by a retrospective chart review of 30 children showing 80% achieved clinical remission. Cablivi should be started with plasma exchange therapy, beginning with an 11mg intravenous (IV) bolus before the first exchange, followed by daily 11mg subcutaneous (SC) doses after each exchange and continuing for 30 days beyond the last exchange, with possible extension up to 28 days if disease persists. The most common side effects are nose bleeds, headache and gum bleeding. Full prescribing information is here.
New 2-Gram Fibryga Kit for Enhanced Bleeding Management Approved
On Jan. 7, 2026, the FDA approved Octapharma USA’s new 2-gram presentation of Fibryga® [Fibrinogen (Human)] Lyophilized Powder for Reconstitution for the treatment of acquired fibrinogen deficiency (AFD) in bleeding patients. This expanded option, which joins the existing 1-gram kit, includes 100mL of Water for Infusion and the nextaro® reconstitution device, enabling faster preparation and greater dosing flexibility in urgent clinical settings. Fibryga is the first virus-inactivated, human plasma-derived fibrinogen concentrate approved for AFD in the US, offering a standardized alternative to cryoprecipitate. The updated prescribing information may be found here.
FDA Approvals in 2025: Issues Document
In 2025, the US Food and Drug Administration (FDA) authorized 58 novel approvals, predominantly comprised specialty drugs, orphan drugs and breakthrough therapies. Among the approvals were 42 specialty drugs and 16 traditional drugs. This document provides a brief overview of the FDA’s approval activity in 2025.