The approval of Doptelet was based on results from the ADAPT-1 (N=231) and ADAPT-2 trials (N=204), 2 identically-designed multicenter, randomized, double-blind, placebo-controlled studies.
Andexxa, a recombinant modified human Factor Xa (FXa) protein, was approved under the FDA's Accelerated Approval pathway.
The safety and efficacy of LentiGlobin were evaluated in 2 independent 2-year clinical studies: HGB-204 (Northstar, N=18), an open-label, single-dose, nonrandomized, multicenter Phase 1/2 study in patients with TDT, and HGB-205, an ongoing open-label, single-dose, nonrandomized, single-center Phase 1/2 study in patients with TDT and severe sickle cell disease.
The trial enrolled 152 patients with hemophilia A who were previously treated, either on-demand or as prophylaxis, with factor VIII therapy.
Complete reversal was seen within 4 hours in the majority of patients, as measured by ecarin clotting time (ECT 82%) or diluted thrombin time (dTT 99%). In addition, there was a low late of thrombotic events and no new safety events were reported.
Safety and efficacy of Vonvendi with or without recombinant factor VIII (FVIII) treatment in elective surgical procedures in patients aged ≥18 years diagnosed with severe von Willebrand disease, was established in the Phase 3 trial.
The recall notification is due to a potential risk of breakage of the glass vials during transport because of a change in the secondary packaging configuration.
Using Medicaid claims from 5 states (1999-2010) and Medicare claims for dual-enrollees, study authors analyzed the first concomitant use of clopidogrel and 1 of 10 selected NSAIDs (ibuprofen, celecoxib, naproxen, rofecoxib, meloxicam, diclofenac, indomethacin, valdecoxib, nabumetone, etodolac) after a 1-year baseline period.
ANNEXA-4 is a global, single-arm, open-label clinical trial designed to evaluate andexanet alfa in patients who present with an acute major bleed while receiving apixaban, rivaroxaban, edoxaban or enoxaparin.
The myPKFiT software can be used to generate Advate dosage amount and frequency recommendations for routine prophylaxis for an individual patient 16yrs of age and older and body weight of 45kg or greater.
The primary endpoint was the number of patients who required no platelet transfusion priory to primary invasive procedure and no rescue therapy for bleeding for 7 days post-procedure.
Rebinyn, a recombinant DNA-derived coagulation Factor IX concentrate with an extended half-life, was approved by the Food and Drug Administration (FDA) in May 2017.
After completion of the initial bevacizumab treatment cycle, there was a significant reduction in epistaxis severity scores and RBC transfusion requirements.
Fitusiran, an RNAi therapeutic targeting antithrombin (AT), is designed to lower levels of AT in order to further sufficient thrombin generation to restore hemostasis and prevent bleeding.
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.