Experimental Treatments for Hemophilia

Aminocaproic acid is an antifibrinolytic medication that can help to stabilize blood clots. It is not formally approved as a treatment for hemophilia, but it may be used off-label in some situations to help control bleeding in hemophilia patients, including to manage bleeding in the mouth, such as during tooth extractions. It is available in the form of oral tablets or solution.

CRISPR/Cas9 is a gene-editing technology that can be used to alter the genetic sequence of a specific gene inside a cell. Treatments using this approach have shown promise in preclinical models of both hemophilia A and hemophilia B, and several companies are working to develop therapies for hemophilia based on CRISPR/Cas9.

Fitusiran is an investigational therapy that’s being developed by Sanofi to prevent bleeding episodes in adults and adolescents with hemophilia A or B, with or without inhibitors. It is administered once monthly via a subcutaneous, or under-the-skin, injection.

Giroctocogene fitelparvovec is an experimental gene therapy currently being developed by Pfizer for the treatment of hemophilia A. Given as a one-time intravenous or into-the-vein, infusion, it aims to reduce the risk of bleeds in hemophilia patients.

Marstacimab is an antibody-based therapy that Pfizer is developing as a prophylactic treatment to prevent or reduce the frequency of bleeding episodes in people with hemophilia A or B. The therapy is administered once weekly via an under-the-skin (subcutaneous) injection.

Mim8 (denecimig) is an investigational therapy being developed by NovoNordisk to prevent or reduce the frequency of bleeding episodes in hemophilia A patients with or without inhibitors. It is given as an under-the-skin, or subcutaneous, injection.

SPK-8011 is an experimental gene therapy being developed by Spark Therapeutics for the treatment of hemophilia A. Given as a one-time intravenous or into-the-vein, infusion, it aims to reduce the risk of bleeds in hemophilia patients.

TRM-201 (rofecoxib) is an oral nonsteroidal anti-inflammatory drug (NSAID) being developed by Tremeau Pharmaceuticals to treat hemophilic arthropathy, a degenerative joint disease that can occur due to recurrent joint bleeds in people with hemophilia.