California center is first in state to offer Hympavzi for hemophilia B
FDA approved therapy last year to reduce bleeding frequency
The Loma Linda University Health (LLUH) Hemophilia Treatment Center has become the first clinic in California offering Hympavzi (marstacimab) for people with hemophilia B.
“For the first time, patients with hemophilia B in Southern California have access to a therapy that is not just effective, but also drastically improves their daily lives,” Akshat Jain, MD, director of the Hemophilia Treatment Center at LLUH, said in a news story from the clinic.
Hemophilia B is caused by mutations that disrupt the activity of factor IX (FIX), a clotting protein. Absent or low FIX activity leads to frequent and easy bleeding, the hallmark symptoms of hemophilia.
For many years, the mainstay treatment for hemophilia B has been replacement therapies, where a version of the FIX protein is administered to help prevent or control bleeds. These treatments can be effective, but they’re often inconvenient for patients because they have to be administered intravenously on a regular basis. Receiving regular infusions into a vein can cause problems with vein health and make it difficult for patients to hold certain jobs, engage in some activities, and traveling, according to LLUH.
What makes Hympavzi different from other hemophilia treatments?
Hympavzi, which is marketed by Pfizer, offers an alternative method of administration — by once-weekly, subcutaneous, or under-the-skin, injections. Also, rather than delivering a version of the FIX protein, Hympavzi blocks the activity of tissue factor pathway inhibitor, a protein that normally helps prevent blood clotting. By blocking the protein, Hympavzi can promote clotting in people with hemophilia B in a way that bypasses the need for FIX.
“This marks a monumental shift in treatment, allowing patients to take control of their health in a way that was not possible before,” Jain said.
The U.S. Food and Drug Administration approved Hympavzi last October as a preventive treatment to reduce the frequency of bleeding episodes in hemophilia B patients, ages 12 and older, who don’t have FIX inhibitors, that is, neutralizing antibodies that can lower the efficacy of standard replacement therapies.
The approval, which also covered Hympavzi for people with hemophilia A without FVIII inhibitors, was based on clinical trial data that showed the once-weekly injection therapy reduced the risk of bleeds over standard therapies.
The first hemophilia B patient at LLUH to receive Hympavzi is a 15-year-old boy who’s been on the therapy for nearly two months. According to LLUH, the youth was “previously dependent on regular [intravenous] infusions,” but “he can now manage the condition with ease, no longer needing to endure repeated needle sticks.”
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