Pfizer has received approval from the US Food and Drug Administration (FDA) for an expanded indication of Hympavzi (marstacimab-hncq) for the treatment of haemophilia A or B.
The approval covers patients aged 12 years and older with inhibitors, as well as children aged six to 11 years, with or without inhibitors.
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Hympavzi is approved for routine prophylaxis to prevent or decrease bleeding episodes in US adults and children aged six years and above with congenital factor VIII or IX deficiency, with or without corresponding inhibitors.
Hympavzi is administered as a once-weekly subcutaneous injection and does not require ongoing treatment-related laboratory monitoring.
Haemophilia, which often presents in early childhood, is characterised by impaired blood clotting. This increases the risk of painful bleeding, especially in the joints, which can lead to joint damage and scarring.
Phase III BASIS trial results supported Hympavzi approval in adults and adolescents with inhibitors, showing a 93% reduction in mean treated annualised bleeding rate compared to on-demand intravenous agents.
Interim data from the Phase III BASIS KIDS trial in children aged six to 17 years demonstrated improved bleeding outcomes versus historical controls.
Reported adverse reactions included injection site reaction, headache, fever, joint pain, diarrhoea, itching, and rash. Two thromboembolic events occurred among 259 patients in an open-label extension.
Thromboembolic events, hypersensitivity, embryofetal toxicity, and laboratory anomalies are highlighted in the Warnings and Precautions section of the US label.
Hympavzi’s application underwent FDA priority review and received breakthrough therapy designation for younger paediatric patients with haemophilia B, expediting the review process.
Pfizer US commercial division chief commercial officer and executive vice president Aamir Malik said: “With this expanded approval, we believe Hympavzi can become a transformative option and meet a significant medical need for people living with haemophilia A or B with or without inhibitors aged six years and older.
“Particularly for children ages six to 11 with haemophilia B who will now, for the first time, have a subcutaneous non-factor treatment available.
“This milestone represents the latest step in Pfizer’s more than 40-year commitment to advancing care and quality of life for people living with haemophilia, which began with the introduction of recombinant therapies and has evolved with the introduction of this once-weekly subcutaneous treatment.”
Earlier this month, Pfizer and Chai Discovery agreed on a licensing deal enabling the former to incorporate Chai’s AI platform into its drug discovery operations.
