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WHO Updates Essential Medicines List to Expand Access to Modern Hemophilia Treatments
09/30/2025
Grace Taylor, MS, MA
The World Health Organization (WHO) has updated its Essential Medicines List to prioritize modern, safer therapies for hemophilia and von Willebrand Disease, advancing global access and aligning policy with current standards of care.
The World Health Organization (WHO) has updated its Essential Medicines List to prioritize modern, safer therapies for hemophilia and von Willebrand Disease, advancing global access and aligning policy with current standards of care.
The World Health Organization...
09/30/2025
First Report Managed Care
News
DTX101 Gene Therapy Ends Development, Offers Key Lessons for Future Hemophilia B Treatment Strategies
09/16/2025
Grace Taylor, MS, MA
Early-phase trials of the DTX101 gene therapy in hemophilia B showed manageable safety but insufficient efficacy, leading to program discontinuation while offering critical insights for future gene therapy development.
Early-phase trials of the DTX101 gene therapy in hemophilia B showed manageable safety but insufficient efficacy, leading to program discontinuation while offering critical insights for future gene therapy development.
Early-phase trials of the DTX101...
09/16/2025
First Report Managed Care
News
Validated Tool Offers Structured Measure of Hemophilia Patients’ Device Preferences
09/16/2025
Grace Taylor, MS, MA
A new study confirms the Hemophilia Device Handling and Preference Assessment (HDHPA) as the first validated tool to capture patient and caregiver perspectives on treatment device preferences.
A new study confirms the Hemophilia Device Handling and Preference Assessment (HDHPA) as the first validated tool to capture patient and caregiver perspectives on treatment device preferences.
A new study confirms the...
09/16/2025
First Report Managed Care
News
High Health Care Burden Among People With Hemophilia A Experiencing Bleeding or Joint Complications
09/10/2025
Juliet Gallagher
New real-world evidence shows that people with hemophilia A who experience bleeding or joint complications face significantly higher health care utilization and comorbidity burden.
New real-world evidence shows that people with hemophilia A who experience bleeding or joint complications face significantly higher health care utilization and comorbidity burden.
New real-world evidence shows...
09/10/2025
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News
FDA Expands Approval of Alhemo for Hemophilia A and B Without Inhibitors
08/14/2025
Juliet Gallagher
Novo Nordisk has announced the US Food and Drug Administration (FDA) approval of Alhemo (concizumab-mtci) for use as a once-daily subcutaneous prophylactic treatment in people 12 years and older with hemophilia A or B (HA/HB) without...
Novo Nordisk has announced the US Food and Drug Administration (FDA) approval of Alhemo (concizumab-mtci) for use as a once-daily subcutaneous prophylactic treatment in people 12 years and older with hemophilia A or B (HA/HB) without...
Novo Nordisk has announced the...
08/14/2025
First Report Managed Care
News
Roctavian Demonstrates Sustained Efficacy and Safety in Patients With Hemophilia A
08/04/2025
Grace Taylor, MS, MA
Five-year data from BioMarin’s phase 3 trial reveals Roctavian’s long-term efficacy, safety, and quality-of-life benefits for adults with severe hemophilia A.
Five-year data from BioMarin’s phase 3 trial reveals Roctavian’s long-term efficacy, safety, and quality-of-life benefits for adults with severe hemophilia A.
Five-year data from BioMarin’s...
08/04/2025
First Report Managed Care
News
Study Confirms Long-Term Safety and Efficacy of AAV Gene Therapy in Hemophilia B
07/22/2025
Grace Taylor, MS, MA
A 13-year follow-up study shows that a single dose of adeno-associated virus (AAV) gene therapy offers durable factor IX expression and sustained clinical benefits in patients with severe hemophilia B.
A 13-year follow-up study shows that a single dose of adeno-associated virus (AAV) gene therapy offers durable factor IX expression and sustained clinical benefits in patients with severe hemophilia B.
A 13-year follow-up study shows...
07/22/2025
First Report Managed Care
News
Patient-Reported Pain Reduction Offers New Path to Predict Treatment Success in Hemophilia
07/08/2025
Juliet Gallagher
A new post hoc analysis from the PERSEPT 1 trial offers compelling evidence supporting the use of eptacog beta for managing acute pain during bleeding episodes (BEs) in patients with hemophilia A or B with inhibitors (PwHABI). The data...
A new post hoc analysis from the PERSEPT 1 trial offers compelling evidence supporting the use of eptacog beta for managing acute pain during bleeding episodes (BEs) in patients with hemophilia A or B with inhibitors (PwHABI). The data...
A new post hoc analysis from the...
07/08/2025
First Report Managed Care
News
Pfizer Reports Strong Phase 3 Results for Hympavzi in Hemophilia A and B With Inhibitors
07/01/2025
Grace Taylor, MS, MA
Pfizer’s hympavzi achieved a 93% reduction in bleeding episodes in a phase 3 trial for patients with hemophilia A and B with inhibitors, offering a promising once-weekly alternative to on-demand therapies.
Pfizer’s hympavzi achieved a 93% reduction in bleeding episodes in a phase 3 trial for patients with hemophilia A and B with inhibitors, offering a promising once-weekly alternative to on-demand therapies.
Pfizer’s hympavzi achieved a 93%...
07/01/2025
First Report Managed Care
News
Emicizumab Shows Promise for Acquired Hemophilia A in Real-World Settings
06/24/2025
Grace Taylor, MS, MA
A real-world study shows that emicizumab offers safe and effective outpatient bleeding prophylaxis for patients with acquired hemophilia A, even amid complex comorbidities.
A real-world study shows that emicizumab offers safe and effective outpatient bleeding prophylaxis for patients with acquired hemophilia A, even amid complex comorbidities.
A real-world study shows that...
06/24/2025
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