FDA Approves First Treatment for Rare Blood Disease

The U.S. Food and Drug Administration approved caplacizumab-yhdp injection as the first treatment specifically indicated for the treatment of adults with acquired thrombotic thrombocytopenic purpura (aTTP) in combination with plasma exchange and immunosuppressive therapy.

The decision was based on results of the HERCULES study, which included 145 patients who were randomized to receive caplacizumab-yhdp or placebo. Participants received an initial intravenous dose followed by daily subcutaneous injections for up to 6 months. Patients in both groups also received plasma exchange and immunosuppressive therapy.

Patients treated with caplacizumab-yhdp experienced a significantly shorter time to platelet count response (primary endpoint) compared with the placebo group (hazard ratio = 1.55; 95% CI, 1.10-2.20; P=0.01). Caplacizumab-yhdp also resulted in a significant reduction of events in a composite endpoint of aTTP-related death, recurrence of aTTP, or a major thromboembolic event during treatment compared with the placebo group (12.7% vs. 49.3%; P<0.0001).

The proportion of patients with a recurrence of aTTP in the overall study period was statistically significantly lower in the caplacizumab-yhdp group (13%) compared with the placebo group (38%).

Common adverse events associated with caplacizumab-yhdp include bleeding of the nose or gums and headache. The prescribing information for caplacizumab-yhdp warns healthcare providers and patients about the risk of severe bleeding.

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Source: FDA