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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
First Report Managed Care
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
First Report Managed Care
News
Eptacog Beta Effective for Severe Bleeds in Hemophilia With Inhibitors
06/18/2025
Grace Taylor, MS, MA
An analysis of phase 3 trial data shows that eptacog beta safely and effectively controls severe bleeding episodes in hemophilia with inhibitors.
An analysis of phase 3 trial data shows that eptacog beta safely and effectively controls severe bleeding episodes in hemophilia with inhibitors.
An analysis of phase 3 trial...
06/18/2025
First Report Managed Care
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