Pfizer has received the European Commission (EC) marketing authorisation for Hympavzi (marstacimab) for treating a type of haemophilia A or B, without inhibitors.
Hympavzi is a rebalancing agent that targets the Kunitz 2 domain of tissue factor pathway inhibitor (TFPI), a natural anticoagulation protein that prevents the formation of blood clots.
The drug is indicated for the routine prophylaxis of bleeding episodes in patients aged 12 years and above weighing at least 35kg with severe haemophilia A without FVIII inhibitors, or haemophilia B without FIX inhibitors.
According to the US drugmaker, Hympavzi is the first and only anti-TFPI drug approved in the European Union (EU) for the treatment of haemophilia A or B.
Also, it is the first haemophilia medicine approved in the EU for administration through a pre-filled, auto-injector pen.
With a once-weekly subcutaneous dosing regimen, Hympavzi provides a convenient treatment option requiring minimal preparation for patients, said Pfizer.
The European regulator granted the marketing authorisation based on results from the Phase 3 BASIS study which evaluated the efficacy and safety of Hympavzi,
In the Phase 3 study, Hympavzi significantly reduced 35% annualised bleeding rate (ABR) for treated bleeds in the 12-month active treatment period.
The study results showed non-inferiority and superiority compared to routine prophylaxis (RP) with FVIII or FIX administered as part of usual care.
Hympavzi showed a safety profile that was consistent with Phase 1/2 results. The most common adverse events were injection site reactions, headache, pruritus, and hypertension.
The EC marketing authorisation is applicable in all 27 European Union (EU) member states, along with Iceland, Liechtenstein, and Norway.
Pfizer chief international commercial officer and executive vice president Alexandre de Germay said: “Hympavzi offers a first-in-class treatment option for people living with haemophilia, a disease that often leads to recurring joint bleeds and can impact daily activities as simple as climbing stairs.
“This approval builds on Pfizer’s more than four-decade commitment to improve the standard of care in haemophilia, and we look forward to delivering this medicine that reduces bleeds as compared to factor prophylaxis and, importantly, requires limited preparation, meeting a key need for eligible patients.”
In a separate development, the UK’s Competition Appeal Tribunal (CAT) has fined Pfizer and Flynn Pharma a total of £69m over unfair pricing of phenytoin sodium capsules.
Previously, the Competition and Markets Authority (CMA) fined Pfizer and Flynn a total of £89.4m in 2016, and £70m in 2022, for unfair pricing of the epilepsy drug.
The tribunal re-made the CMA’s decision, based on separate infringement findings. The CAT found that Pfizer and Flynn abused their strong position in the market by charging excessive prices for a life-saving epilepsy drug, between 2012 and 2016.
