Pfizer’s HYMPAVZI™ Approved by European Commission for Severe Haemophilia A and B
Thursday, November 21, 2024
Pfizer has announced that the European Commission (EC) has granted marketing authorisation for HYMPAVZI™ (marstacimab), its treatment for adolescents and adults aged 12 years and older with severe haemophilia A or B without inhibitors.
HYMPAVZI is the first anti-tissue factor pathway inhibitor (anti-TFPI) approved in the EU for haemophilia A and B. The treatment, delivered through a pre-filled auto-injector pen, offers a once-weekly subcutaneous dosing option with minimal preparation required.
Haemophilia, a rare genetic blood disorder affecting over 800,000 people globally, results from a deficiency in clotting factors—Factor VIII (FVIII) in haemophilia A and Factor IX (FIX) in haemophilia B.
This approval is applicable across all 27 EU member states, as well as Iceland, Liechtenstein, and Norway.
The condition impairs blood clotting, leading to recurring bleeding episodes, often causing joint damage and impacting daily life. Despite advances in treatment, many individuals still require frequent intravenous infusions, which can be time-consuming and challenging to manage.
Pfizer’s HYMPAVZI aims to address these challenges by offering a convenient and effective option. The treatment reduces bleeding episodes and avoids the need for frequent infusions.
The EC’s approval is based on findings from the pivotal Phase 3 BASIS study, which evaluated HYMPAVZI in 116 participants aged 12 and above with severe haemophilia A or B without inhibitors. Over a 12-month active treatment period, HYMPAVZI demonstrated a 35% reduction in annualised bleeding rates compared to routine prophylaxis with FVIII or FIX. These results established both the safety and efficacy of the treatment.
Adverse events observed during the study were consistent with earlier trials and included injection site reactions, headaches, itching, and hypertension. An ongoing extension study has shown continued reductions in bleeding rates with long-term use.
The ongoing BASIS study will provide further insights, particularly for patients with inhibitors, with results expected in 2025. Additionally, Pfizer is conducting research on HYMPAVZI for children aged 1 to 18 years in the BASIS KIDS study.
This approval builds on Pfizer’s extensive work in haemophilia treatment, which spans over four decades. Alongside HYMPAVZI, the company has advanced gene therapy candidates and received recent regulatory approvals for haemophilia B treatments in Europe and the US.
Pfizer’s HYMPAVZI represents a significant step forward in providing a simpler and more accessible treatment option for eligible individuals living with haemophilia.
Source: pfizer.com
