The Treatment Landscape: New Developments in Bleeding Disorders Care

The treatment horizon for bleeding disorders looks remarkably different than it did just a few years ago. From nerve stimulation devices to injections every two months, from experimental pills to designer antibodies, the future of bleeding disorders care is unfolding with possibilities that extend far beyond traditional factor replacement. The popular, “Advancements in Treatments forn Bleeding Disorders,” took a look into the future.

Expanding Beyond Hemophilia

Amy Dunn, MD, Chair of NBDF’s Medical and Scientific Advisory Council (MASAC), began the session by introducing new therapies for non-hemophilia bleeding disorders—a group, she noted, which has been under-represented in advancements in bleeding disorders care.

New developments are on the horizon for not only hemophilia, but also von Willebrand disease (VWD) and Glanzmann's thrombosthenia, conditions that have historically had fewer treatment options.

Emicizumab: Exploring New Applications

Emicizumab (Hemlibra), originally approved to treat hemophilia A with and without inhibitors, is now being studied as a potential treatment for severe VWD. 

"Most people who have severe forms of von Willebrand disease also have quite low factor eight," explained Dunn. "Because von Willebrand protein actually carries factor eight to where it participates in coagulation." While emicizumab is not currently approved to treat VWD, clinical trials are investigating its potential use as a VWD treatment.

Novel Approaches: Neurostimulation Technology

Another new technology explored was OhmBody, a vagus nerve stimulation device, which represents a different approach to treating bleeding disorders. "As it turns out, if you know the right frequencies, you know the right nerves, you can stimulate platelet function,” which are essential for coagulation. Dunn explained. A preliminary study found that people who wore the device for one hour twice a day during their periods were able to reduce their heavy menstrual bleeding by 50%. A large clinical trial will soon be underway to study this more thoroughly.

Glanzmann Thrombasthenia: First Effective Treatment in Trials

For the first time, an effective treatment for Glanzmann thrombasthenia is in phase 2 clinical trials and showing encouraging results. The data—presented at the ISTH 2025 congress—show over 50% reduction in treated bleeds. "I think you're starting to see that these designer antibody therapies can really be designed for many different bleeding disorders," Dunn said.

Extended Half-Life Therapies: Next Generation

Dr. Dunn moved back to treatments for hemophilia, discussing a newer therapy called Altuviiio. 

Altuviiio, an extended half-life therapy for hemophilia A, was designed to last longer in the body. Compared to traditional products, which fall below protective levels in two days or less, Altuviiio can maintain those levels for about seven days. This allows patients to infuse less often while keeping factor levels steady, which is especially helpful for active individuals and children.

"Your treatment center can look at your child's PK and really aim to keep your child's bleeding rate at zero, which is what we all want," she added.

Factor VIII Mimetics: Emerging Options

The speaker shared updates on three new factor VIII mimetic drugs for hemophilia A—Mim8, NXT007, and a third still in development. None are FDA-approved yet, but early data from trials has been reported.

These are  bispecific antibody therapiesThis treatment works as a bridge between factors.. Mim8 may offer practical advantages, with fewer loading doses and a pen-style injector. In trials, it showed notable results: most patients (84%) had no treated bleeds.

NXT007 is smaller and made using antibodies from llamas, which allows for a precise, targeted effect. Its developer is exploring a way to eventually make it orally available—which would represent a significant advance.

What makes NXT007 particularly interesting is that it's being developed with a technology called SNAP, which may eventually allow drugs like this to be taken orally (by mouth)—something that would be a major breakthrough for people with bleeding disorders.

Rebalancing Agents: New Prophylaxis Approach

The speaker discussed three new rebalancing drugs for hemophilia, which are designed to help blood clot better by adjusting the body's natural clotting balance. These fall into two groups: two drugs block something called tissue factor pathway inhibitor (TFPI)—these are concizumab (Alhemo) and marstacimab (Hymphazi)—and siRNA lowers antithrombin levels, called fitusiran (Qfilitia).

Normally, the body starts clotting through a complex process where TFPI quickly turns off part of it, expecting factor VIII and IX to take over. These new drugs block TFPI, keeping clotting signals active longer, or reduce antithrombin, which usually slows clotting, thus rebalancing clotting in people with hemophilia.

Current Approved Options

Three rebalancing agents are now FDA-approved:

• Concizumab is FDA-approved for hemophilia A and B, with or without inhibitors, for people 12 and older. It's given daily by a reusable pen with very tiny needles, and clinical trials are ongoing for younger kids.
• Marstacimab is approved for hemophilia A and B without inhibitors, for 12 and older. It's given once weekly in a single-use pen, with trials underway for kids and possibly inhibitor patients.
• Fitusiran is approved for hemophilia A and B, with or without inhibitors, over age 12. It's given every other month via single-use pen, but requires careful monitoring of antithrombin levels to avoid clot risks.

Important Considerations

All three are treatments designed to prevent bleeds. In the event of a breakthrough bleed, traditional factor products may need to be used., If bleeds do occur, treatment must be carefully managed with guidance from healthcare providers to avoid clotting risks.

For surgery, minor procedures usually don't require stopping these drugs, but major surgery often does, with a return to previous treatments.

Switching between these new agents is complicated, as overlapping effects can raise clot risk. There's limited data, so any changes must be carefully planned with a treatment center.

Looking Forward

The new technologies for treating bleeding disorders continue to advance through clinical trials and FDA approvals, expanding the range of options available to patients and their healthcare providers. These developments represent ongoing progress in addressing the diverse needs of the bleeding disorders community.