In all participants, vector-derived factor IX coagulant activity was sustained, with a mean steady-state factor IX coagulant activity of 33.7 ± 18.5%.
TRM-201 (rofecoxib) is a highly potent COX-2 selective non-steroidal anti-inflammatory drug (NSAID) with a well-established efficacy profile.
Alprolix is a recombinant clotting factor therapy developed using Fc fusion technology to prolong circulation in the body.
Hemlibra is a bispecific factor IXa- and factor X-directed antibody indicated for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adult and pediatric patients with hemophilia A (congenital factor VIII deficiency) with factor VIII inhibitors.
Overall, 63% of bleeding deaths occurred within 12 hours of onset. There was a peak in deaths from postpartum hemorrhage at 2 to 3 hours after childbirth.
BAY94-9027 is designed to prolong FVIII activity in the blood while preserving coagulation activity using site-specific technology in which the Polyethylene glycol molecule is consistently attached to the factor VIII protein at a specific site.
The Company has decided in conjunction with the FDA to suspend further administration of Octagam 10% from these specific production lots.
The FDA has accepted and granted Priority Review to the Biologics License Application (BLA) of emicizumab (Genentech) for the prophylactic treatment of hemophilia A in adults, adolescents and children with factor VIII inhibitors.
In addition to the currently available strengths (250, 500, 1000, 2000 IU), starting in September, Nuwiq will also be supplied in 2500, 3000, and 4000 IU vial strengths.
The Phase 2 TITAN study (n=75) evaluated the safety and efficacy of caplacizumab with standard of care of plasma exchange and immunosuppression.
The primary analysis of the study arm showed a statistically and clinically significant reduction in bleeding by 87% (risk rate [RR] 0.13; P<0.0001] with emicizumab prophylaxis.
Tavalisse is an oral treatment designed to inhibit SYK kinase, a key signaling member in the immune process that leads to platelet destruction in ITP.
Rebinyn, a recombinant DNA-derived coagulation Factor IX concentrate, temporarily replaces the missing coagulation Factor IX in hemophilia B patients.
The Food and Drug Administration (FDA) has granted Fast Track designation to SB-525 (Sangamo Therapeutics) for the treatment of hemophilia A.
Rigel announced its submission of a New Drug Application (NDA) to the Food and Drug Administration (FDA) for fostamatinib for the treatment of patients with chronic and persistent immune thrombocytopenic purpura (ITP).
The interim results from HAVEN 2, after a median of 12 weeks of treatment, showed that prophylaxis with emicizumab is associated with clinically meaningful reduction in the number of bleeds over time.
Commenting on the study's results, lead author Robert J. Adams, MD, said, "Having sickle cell disease did not adversely affect any of the indicators we measured."
While the GHR tests may provide genetic risk information, they cannot determine a person's overall risk for developing a disease, as other factors beyond genetics (ie, environment, lifestyle) may also influence risk.
Patients taking apixaban showed significantly less intracranial hemorrhage (0.33% per year) vs. patients taking warfarin (0.80% per year) no matter the type and location.
Fifty-two (10.9%) of the patients who experienced an ischemic event, and 41 (17.7%) who suffered experieced a bleeding event, died.
Nipride RTU is approved for immediate blood pressure reduction and also carries a Boxed Warning describing the possibility of excessive hypotension and cyanide toxicity.
The Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to the investigational gene therapy, AMT-060 (uniQure), for the treatment of severe hemophilia B.
LFB S.A. announced that the FDA has accepted for review the Biologic License Application (BLA) of its recombinant Coagulation Factor VIIa (eptacog beta, activated) for the potential treatment of congenital hemophilia A or B in adolescents and adults with inhibitors.