The US big pharma company Pfizer has been granted marketing authorization for its hemophilia A and B treatment, HYMPAVZI (marstacimab), by the European Commission based on positive Phase 3 study results. In the EU, the drug is the first once-weekly, subcutaneous treatment for people with severe hemophilia B, and the first that can be administered via a pre-filled, auto-injector pen. The marketing authorization in the EU follows the FDA’s regulatory approval from October.
Over 800,000 people worldwide live with the rare genetic blood disease hemophilia, which is caused by a deficiency of the clotting factor FVIII in people with hemophilia A and clotting factor FIX in people with hemophilia B. The blood of affected persons cannot clot properly, meaning that there is an increased risk of internal bleeding inside the joints and other organs when injury occurs. Currently, people with hemophilia have to receive regular intravenous infusions of FVIII or FIX, sometimes several times a week.
“There is a considerable treatment burden associated with the standard-of-care options for hemophilia A and B, including time-consuming preparation and administration of infusions and injections potentially causing missed doses and an increased risk of bleeding,” said Laurent Frenzel, Head of the Hemophilia Treatment and Research Center at the Necker-Enfants Malades Hospital, Paris, in a press release.
“HYMPAVZI is a significant advancement for eligible patients in that it may provide bleed prevention as well as once-weekly subcutaneous administration via a pre-filled pen.”
The European Commission’s marketing authorization resulted from positive efficacy and safety results from the pivotal Phase 3 study, BASIS. HYMPAVZI was tested in adults and adolescents aged 12 years or older with severe hemophilia A or B without inhibitors. The results showed that the drug lowered the number of times people had to be treated for bleeds: The annual bleeding rate for treated bleeds was reduced by 35%. This, says Pfizer, demonstrated “non-inferiority and superiority compared to routine prophylaxis with FVIII or FIX administered as part of the usual care.”
Speaking of the European Commission’s decision, Alexandre de Germay, Chief International Commercial Officer and Executive Vice President of Pfizer, said in a press release: “HYMPAVZI offers a first-in-class treatment option for people living with hemophilia, 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 hemophilia, and we look forward to delivering this medicine that reduced bleeds as compared to factor prophylaxis and, importantly, requires limited preparation, meeting a key need for eligible patients.”
