Hemophilia A is an inherited bleeding disorder caused by deficiency or dysfunction of clotting factor VIII, leading to impaired coagulation and prolonged bleeding. Clinical severity ranges from mild to severe and can include spontaneous joint and muscle hemorrhage in severe disease. It is the most common form of hemophilia.
The European Commission expanded Hympavzi approval to patients 12 and older with hemophilia A or B with inhibitors. Phase 3 data showed a 93% reduction in mean treated annualized bleeding rate versus on-demand therapy.
Global orphan drug sales are forecast to reach $409 billion by 2032, representing one-fifth of prescription drug sales, with Johnson & Johnson and Argenx leading the market amid regulatory uncertainty.
Pfizer reported positive Phase 3 trial results for BRAFTOVI combination therapy in metastatic colorectal cancer and received FDA Priority Review for HYMPAVZI expansion to hemophilia patients including children.
CSPC Pharmaceutical Group has received approval from China's National Medical Products Administration to begin clinical trials for three drug candidates: a hemophilia A biosimilar, a long-acting pain medication, and an injection designated SYH9089.
| NCT ID | Title | Status | Phase |
|---|---|---|---|
| NCT07582276 |
A Multi-institution Prospective Assessment of Bone Health in Patients With Severe Hemophilia A on Factor VIII vs Factor Mimetic Prophylaxis (Efa Emi Bone Health Study) |
NOT_YET_RECRUITING | |
| NCT07539402 |
Searching Patterns In the Robustness of Immunological FVIII Tolerance |
NOT_YET_RECRUITING | |
| NCT07523399 |
Joint Health, Balance and Quality of Life in Adults With Hemophilia A |
RECRUITING | |
| NCT07416604 |
A Clinical Study to Evaluate the Effects of NXT007 Compared to Emicizumab Prophylaxis in People With Hemophilia A |
RECRUITING | PHASE3 |
| NCT07416526 |
A Clinical Study to Evaluate the Effects of NXT007 Compared to Factor VIII Prophylaxis in Participants With Hemophilia A |
RECRUITING | PHASE3 |
| NCT07414511 |
Hemophilia A Research Program |
RECRUITING | |
| NCT07314983 |
Long-term Anticoagulation in a Patient With Severe Hemophilia A |
RECRUITING | |
| NCT07226206 |
A Gene Therapy Study of SPK-8011QQ in Adults With Severe or Moderately Severe Hemophilia A |
RECRUITING | PHASE1/PHASE2 |
| NCT07190118 |
Effects of Emicizumab Treatment in Patients With Hemophilia A |
NOT_YET_RECRUITING | |
| NCT07116993 |
Effects of Ultrasound and Infrared on Joint Pain and Movement in Hemophilia A Patients. |
COMPLETED | NA |
