European Commission Approves Pfizer’s Haemophilia Treatment

According to the US Centers for Disease Control and Prevention, one in every 5,000 male births is affected by inherited haemophilia.

Pfizer is responsible for manufacturing Haemophilia treatment and has been granted marketing authorisation from the European Commission to expand its portfolio.

The medical company is looking to include patients aged 12 and older who weigh at least 35kg into its haemophilia treatment range.

What is haemophilia?

According to the NHS, haemophilia is a rare condition that affects the way the blood clots, meaning that the person bleeds more easily.

Haemophilia is a condition that people are born with and is caused by a lack of protein, called clotting factor, that helps the blood to clot.

Caused by an altered gene, this inherited disease tends to show symptoms more severely in men than in women.

The NHS states there are two types of this disease, depending on the type and amount of clotting factor protein in the blood.

Haemophilia A is caused by a lack of clotting factor eight, while Haemophilia B is caused by a lack of clotting factor nine.

Both have the same symptoms (bleeding for a long time, easily bruised and joint pain/stiffness) and are generally incurable and are treated with different medicines.

“For people living with haemophilia with inhibitors, recurring bleeding episodes can lead to damaged joints and introduce real limitations and disruptions to everyday life,” says Alexandre de Germay, Chief International Commercial Officer and Executive Vice President, Pfizer.

“This approval brings a once-weekly medicine to the EU that meets a critical need for patients who face a treatment journey that can be complex and challenging with limited options available today, representing the latest step in Pfizer’s more than 40-year commitment to advancing care for people living with haemophilia. 

“We look forward to working with regulators globally to continue bringing HYMPAVZI to those who can benefit from it.” 

Pfizer’s haemophilia treatment

Pfizer has been given the go-ahead to expand its HYMPAVZI (marstacimab) haemophilia treatment to include patients 12 years of age and older weighing at least 35 kg with haemophilia A (congenital factor VIII deficiency) with FVIII inhibitors or haemophilia B (congenital factor IX deficiency) with FIX inhibitors.

HYMPAVZI offers a combination of superior bleed protection compared to on-demand treatment that is well-tolerated with a straightforward, once-weekly subcutaneous injection administration that does not require routine treatment-related lab monitoring for this difficult-to-treat inhibitor patient population 12 years of age and older.

Inhibitors significantly reduce treatment options for people living with haemophilia by neutralising factor replacement therapies and making them ineffective. 

Affecting around 20% of people with haemophilia A and 3% with haemophilia B, these antibodies prevent therapies from stopping or preventing bleeding episodes, particularly in patients who do not respond to immune tolerance induction therapy.

“Inhibitors present a substantial challenge for people living with haemophilia as they neutralise traditional factor replacement therapies, in turn limiting treatment options and leaving patients vulnerable to uncontrolled bleeding episodes,” says Dr Laurent Frenzel, Head of the haemophilia Treatment and Research Centre at the Necker-Enfants Malades Hospital (Paris Cité). 

“The approval of HYMPAVZI offers adults and adolescents in the EU a once-weekly subcutaneous option that has demonstrated the ability to reduce bleeding episodes and maintain bleed reduction based on observation to date in a long-term extension study.” 

A successful phase three trial

The indication extension is supported by Phase 3 BASIS trial results, which showed HYMPAVZI significantly reduced bleeding rates in patients aged 12 and older with severe haemophilia A or moderately severe to severe haemophilia B with inhibitors. 

Treatment achieved a 93% reduction in annualised bleeding rates compared with on-demand therapy and demonstrated superiority across all key bleeding-related secondary endpoints, including spontaneous, joint and target joint bleeds. 

Long-term extension data also showed bleeding rates remained consistently low for up to 53 months of treatment.

The safety profile aligned with earlier studies, with injection site reactions, headache, itching, hypertension and rash among the most commonly reported side effects, while thrombosis was the most serious adverse event observed. 

The marketing authorisation applies across all 27 EU member states, as well as Iceland, Liechtenstein and Norway. Separately, the US FDA has granted Priority Review to an application seeking to expand HYMPAVZI’s use to younger patients and those with inhibitors.