FDA

Cablivi OK’d for acquired thrombotic thrombocytopenic purpura in pediatric patients

January 9, 2026

On January 5, 2026, FDA expanded the approval of Cablivi (caplacizumab-yhdp), a von Willebrand factor (vWF)-directed antibody fragment, to include the treatment pediatric patients 12 years of age and older with acquired thrombotic thrombocytopenic purpura (aTTP), in combination with plasma exchange and immunosuppressive therapy.

Efficacy

Efficacy in the treatment of pediatric patients with aTTP was evaluated in 30 patients (≤18 years of age) from an observational, retrospective chart review study (NCT05263193). Median age at Cablivi initiation was 15 years (range, 2-18 years). Twenty-one patients (70%) were older than 12 years of age and 9 patients (30%) were less than or equal to 12 years old at aTTP diagnosis. Median body weight was 72.4 kg (range, 9-130 kg) at initiation. Median study duration was 0.31 years and median duration of treatment was 31 days. Among the 30 patients, 29 received plasma exchange treatment that began after Cablivi was initiated and among them the median duration of plasma exchange treatment was 6 days. Most of the patients (90%) received Cablivi adult dose at first initiation. Major efficacy outcomes included clinical remission, time to platelet count response, refractory disease, and disease recurrence.

Results showed 80% (95% confidence interval, 62.7-90.5) of patients achieved clinical remission, defined as a platelet count of at least 150×10⁹/L and a lactate dehydrogenase level less than 1.5 times the upper limit of normal for 30 days or more after cessation of therapeutic plasma exchange.

Safety

The most commonly reported events were epistaxis in 4 (13.3%) patients and tachycardia in 4 (13.3%) patients. One serious bleeding adverse reaction (hemorrhage urinary tract) was reported. The adverse reaction profile in pediatric patients 12 years and older with aTTP was consistent with that in adults.

Sources:

Cablivi (caplacizumab-yhdp) [package insert]. Food and Drug Administration. https://www.accessdata.fda.gov/drugsatfda_docs/label/2025/220449s000lbl.pdf Revised December 2025. Accessed January 7, 2026.

FDA approves therapy for rare blood disorder in pediatric patients 12 years and older. [News release]. 2026. https://www.fda.gov/drugs/news-events-human-drugs/fda-approves-therapy-rare-blood-disorder-pediatric-patients-12-years-and-older