Pfizer gains EU approval for Hympavzi, Phase III shows 93% bleed reduction
Pfizer won EU marketing authorisation for weekly subcutaneous Hympavzi to treat haemophilia A or B with inhibitors in patients aged 12+ weighing at least 35 kg, based on Phase III BASIS data showing a 93% reduction in annualised bleeding rate. The FDA has granted priority review for pediatric expansion, decision expected Q2 2026.
1. EU Approval for Hympavzi
Pfizer secured marketing authorisation from the European Commission to expand Hympavzi’s indication to haemophilia A or B patients with inhibitors aged 12 years and above who weigh at least 35 kg. This approval encompasses all 27 EU member states plus Liechtenstein, Norway and Iceland, enabling once-weekly subcutaneous dosing without routine laboratory monitoring.
2. Phase III BASIS Trial Results
The label expansion is underpinned by Phase III BASIS trial data demonstrating a 93% reduction in mean treated annualised bleeding rate compared with on-demand therapy. The trial monitored efficacy and safety over up to 53 months, identifying injection site reactions, headache, pruritus, hypertension and rash as the most common adverse events, while thrombosis was the most serious.
3. FDA Priority Review and Outlook
In the United States, the FDA has granted priority review for the supplemental biologics licence application to extend Hympavzi’s indication to patients aged six years and above with haemophilia A or B with inhibitors. A regulatory decision is expected by the end of Q2 2026, positioning Pfizer for potential US label expansion.