- |||||||||| Obizur (susoctocog alfa) / Takeda, Ipsen
Evaluation of Factor VIII Population Pharmacokinetics and Exposure-Response to Support Dosing of Recombinant Porcine Factor VIII in Patients with Acquired Hemophilia A (Exhibition Hall) - May 17, 2024 - Abstract #ISTH2024ISTH_1701;
P=N/A, P2,
A typical adult patient (70 kg, 65 years, with positive hFVIII and negative pFVIII inhibitors at baseline) had 10.5 dL/h FVIII clearance, 72.8 dL volume of distribution (Vc), and 6.7 h elimination half-life. Patients with bleeding and positive baseline pFVIII inhibitors (n=13) had 10-fold and 2-fold higher median FVIII clearance and Vc, respectively, than those with negative baseline pFVIII inhibitors (n=19; Table 2); patients positive for baseline hFVIII inhibitors had 3.5-fold and 2-fold higher median FVIII clearance and Vc, respectively, than those negative for baseline hFVIII inhibitors (Table 2).
- |||||||||| Obizur (susoctocog alfa) / Takeda, Ipsen
BLEEDING DUET: A CASE OF SIMULTANEOUS ACQUIRED FACTOR VIII DEFICIENCY AND ACQUIRED VON WILLEBRAND SYNDROME (Riverwalk AB; Virtual) - Mar 2, 2024 - Abstract #THSNA2024THSNA_42;
To the authors knowledge, this is the first documented instance of two acquired bleeding disorders that occurred simultaneously in a single individual. Here we present the diagnostic steps taken to identify and then prove the presence of two distinct acquired autoimmune mediated bleeding entities and documented positive longitudinal response to immunosuppression.
- |||||||||| Hemlibra (emicizumab-kxwh) / Roche, Obizur (susoctocog alfa) / Takeda, Ipsen
Journal: Diagnosis and laboratory monitoring of hemophilia A. (Pubmed Central) - Dec 9, 2023
Measurement of rpFVIII requires a 1-stage clotting assay, and measurement of patient FVIII while on emicizumab requires a chromogenic assay that does not contain human FX. Close communication is required between the treating clinicians and the laboratory to ensure that the correct tests are performed while patients are receiving treatments.
- |||||||||| Obizur (susoctocog alfa) / Takeda, Ipsen
REAL-WORLD SAFETY AND EFFECTIVENESS OF RECOMBINANT B-DOMAIN () - Feb 13, 2023 - Abstract #EAHAD2023EAHAD_380;
P=N/A, P2/
Background: Chemoimmunotherapy based on rituximab remains the cornerstone of treating symptomatic patients with Waldenstr Introduction: Recombinant porcine factor VIII (rpFVIII; susoctocog alfa, Obizur
- |||||||||| Hemlibra (emicizumab-kxwh) / Roche
Monitoring of Emicizumab in Acquired Hemophilia A (#227) (foyer) - Feb 8, 2023 - Abstract #GTH2023GTH_307;
Conclusion Modification of coagulation methods allows monitoring of emicizumab, residual FVIII and inhibitor titer in patients with AHA. The monitoring program established here will be helpful for assessing the use of emicizumab in clinical trials and during real-world use.
- |||||||||| Obizur (susoctocog alfa) / Takeda, Ipsen
Long term observation of therapy and outcome in acquired hemophilia A (#122) (foyer) - Feb 8, 2023 - Abstract #GTH2023GTH_306;
However, due to the relatively small sample size, more data needs to be collected. Due to the rarity of AHA and a lack of RCTs, retrospective studies like this make a major contribution to provide more precise prognosis and better aftercare for future patients.
- |||||||||| Obizur (susoctocog alfa) / Takeda, Ipsen
Trial completion, Preclinical: Study of TAK-672 in Participants With Acquired Hemophilia A (clinicaltrials.gov) - Dec 29, 2022
P2/3, N=5, Completed,
Due to the rarity of AHA and a lack of RCTs, retrospective studies like this make a major contribution to provide more precise prognosis and better aftercare for future patients. Active, not recruiting --> Completed
- |||||||||| Obizur (susoctocog alfa) / Takeda, Ipsen
Journal: Correlation research of susceptibility single nucleotide polymorphisms and the severity of clinical symptoms in attention deficit hyperactivity disorder. (Pubmed Central) - Oct 11, 2022
Of the remaining 15 loci, there were 9 SNPs, rs2652511 (SLC6A3 locus), rs1410739 (OBI1-AS1 locus), rs3768046 (TIE1 locus), rs223508 (MANBA locus), rs2906457 (ST3GAL3 locus), rs4916723 (LINC00461 locus), rs9677504 (SPAG16 locus), rs1427829 (intron) and rs11210892 (intron), correlated with the severity of clinical symptoms of ADHD...Our study builds on previous susceptibility research and further investigates the impact of a single SNP on the severity of clinical symptoms of ADHD. This can help improve the diagnosis, prognosis and treatment of ADHD.
- |||||||||| Obizur (susoctocog alfa) / Takeda, Ipsen
Enrollment closed: Study of TAK-672 in Participants With Acquired Hemophilia A (clinicaltrials.gov) - Sep 30, 2022
P2/3, N=5, Active, not recruiting,
This can help improve the diagnosis, prognosis and treatment of ADHD. Recruiting --> Active, not recruiting
- |||||||||| Obizur (susoctocog alfa) / Takeda, Ipsen
Journal: Analytical Performance of Different Laboratory Methods for Measuring Susoctocog-Alfa. (Pubmed Central) - Aug 27, 2022
The results showed that the reagent combinations reaching satisfactory analytical performance are: SynthasIL/human calibrator/deficient plasma+VWF (total recovery 99.4%, inter-laboratory CV 4.04%), SynthasIL/porcine calibrator/deficient plasma+VWF (total recovery 111%, inter-laboratory CV 2.75%) and Chromogenic/ porcine calibrator/deficient plasma+VWF (total recovery 96.6%, inter-laboratory CV 8.32%). This study highlights that the use of porcine standard (when available) and FVIII deficient plasma with VWF should be recommended.
- |||||||||| Obizur (susoctocog alfa) / Takeda, Ipsen
Clinical, Preclinical, Journal: Recombinant porcine factor VIII corrects thrombin generation in vitro in plasma from patients with congenital hemophilia A and inhibitors. (Pubmed Central) - Jun 30, 2022
Neutralizing factor VIII (FVIII) antibodies are a major complication in hemophilia A. Antihemophilic factor VIII (recombinant), porcine sequence (rpFVIII; susoctocog alfa; Baxalta US Inc., a Takeda company) has low cross-reactivity to anti-human FVIII antibodies and can provide functional FVIII activity in the presence of FVIII inhibitors...Recombinant porcine factor VIII demonstrated a dose-dependent correction of thrombin generation and clot formation in vitro, dependent on the anti-porcine FVIII inhibitor titer. Procoagulant responses to rpFVIII occurred in plasma containing FVIII inhibitors.
- |||||||||| Obizur (susoctocog alfa) / Takeda, Ipsen
Analytical performance of different laboratory methods for measuring susoctog-alpha (Exhibition) - May 13, 2022 - Abstract #ISTH2022ISTH_1724;
Conclusion(s): The present study highlights the impact of the calibrator and of the deficient plasma used in the assay on the accuracy of the results. The use of porcine standard (when available) and FVIII deficient plasma with vWF is recommended.
- |||||||||| Obizur (susoctocog alfa) / Takeda, Ipsen, Rituxan (rituximab) / Biogen, Zenyaku Kogyo, Roche
Journal: Acquired hemophilia A in the setting of dual anticoagulation therapy and lupus anticoagulant: a case report. (Pubmed Central) - May 10, 2022
The use of porcine standard (when available) and FVIII deficient plasma with vWF is recommended. Diagnosis of acquired hemophilia A can be confounded by other causes of abnormal coagulation studies and may require specialized testing, such as a chromogenic Bethesda assay, to confirm the presence of a factor VIII inhibitor.
- |||||||||| The in vitro procoagulant effect of recombinant porcine factor VIII in patients with inherited haemophilia A and inhibitors () - May 7, 2022 - Abstract #WFH2022WFH_628;
Introduction: Susoctocog alfa (Obizur; Takeda, Japan) is a B domain-deleted recombinant porcine FVIII (r-pFVIII) molecule...Increasing concentrations of aPCC (Feiba; Takeda, Japan) 0-12.5-25-50-100 U/kg; rFVIIa (NovoSeven; NovoNordisk, Danemark) 45-90-180-200270 μg/kg and r-pFVIII 6-12.5-25-50-100-200 IU/dL were tested... TG assay may help to evaluate the efficacy of r-pFVIII in PWIHAI, in particular in those receiving prophylaxis with emicizumab, in whom the detection of r-pFVIII inhibitors is impossible with routine laboratory methods.
- |||||||||| NovoSeven (eptacog alfa) / Novo Nordisk, Obizur (susoctocog alfa) / Takeda, Ipsen
Journal: Time is Blood: The Impact of Diagnostic Delays on Acquired Hemophilia A. (Pubmed Central) - Mar 29, 2022
Four of these five patients died directly from bleeding complications. Conclusions Based on our study findings and review of the literature, we propose an algorithm to potentially aid in the early diagnosis and treatment of AHA in emergency and non-specialized settings.
- |||||||||| Obizur (susoctocog alfa) / Takeda, Ipsen
Enrollment open, Preclinical: Study of TAK-672 in Participants With Acquired Hemophilia A (clinicaltrials.gov) - Dec 23, 2021
P2/3, N=5, Recruiting,
This study provides insights into the use of rpFVIII as part of a hemostatic treatment regimen in real-life clinical practice. Not yet recruiting --> Recruiting
- |||||||||| Obizur (susoctocog alfa) / Takeda, Ipsen
Preclinical, Enrollment change: BAX 802 in CHA With Inhibitors (clinicaltrials.gov) - Oct 20, 2021
P3, N=8, Terminated,
Thus, good hemostasis can be achieved with rpFVIII within the first days after surgery; other bypassing agents should be used if longer clotting factor correction is required. N=14 --> 8
- |||||||||| Obizur (susoctocog alfa) / Takeda, Ipsen
Preclinical, Trial completion date, Trial initiation date, Trial primary completion date: Study of TAK-672 in Participants With Acquired Hemophilia A (clinicaltrials.gov) - Jul 1, 2021
P2/3, N=5, Not yet recruiting,
Recruiting --> Completed Trial completion date: Mar 2022 --> Nov 2022 | Initiation date: Mar 2021 --> Nov 2021 | Trial primary completion date: Mar 2022 --> Nov 2022
- |||||||||| Obizur (susoctocog alfa) / Takeda, Ipsen
Journal: Characterization of a spontaneous cyst-forming strain of Toxoplasma gondii isolated from Tokachi subprefecture in Japan. (Pubmed Central) - Jun 22, 2021
Although virulence of Obi1 strain is significantly lower than that of ME49, nucleotide sequences analyses revealed that genes of virulence factors such as Gra15, Rop5, 16, 17, and 18 in Obi1 strain were 100% identical to those in the type II strain. Thus, characterization of a newly isolated strain, Obi1, is crucial to clarify the development of toxoplasmosis in both humans and animals.
- |||||||||| Obizur (susoctocog alfa) / Takeda, Ipsen
Preclinical, Journal: Acquired haemophilia and haemostatic control with recombinant porcine FVIII: case series. (Pubmed Central) - Jun 1, 2021
This case series demonstrates that recombinant porcine FVIII is efficacious to treat acute bleeds in acquired haemophilia, including in those who are refractory to bypassing agents. Doses of rpFVIII were able to be titrated based on FVIII level and clinical response.
- |||||||||| Obizur (susoctocog alfa) / Takeda, Ipsen
Preclinical, Journal: Recombinant porcine FVIII for bleed treatment in acquired hemophilia A: findings from a single-center, 18-patient cohort. (Pubmed Central) - May 20, 2021
Recombinant porcine FVIII (rpFVIII: Obizur, Baxter, Deerfield, IL) was approved by the US Food and Drug Administration (FDA) for bleed treatment in AHA in 2014, and has the advantage of laboratory monitoring of FVIII activity levels and known hemostatic efficacy in the presence of anti-human FVIII inhibitors and after failure of BPAs...We also present data showing lower total rpFVIII use for initial bleed resolution when rpVIII is used upfront, as compared with use as rescue therapy. We validated our dosing algorithm, which uses much lower than FDA-recommended doses with 14 more patients than in our previously reported patient series.
- |||||||||| Obizur (susoctocog alfa) / Takeda, Ipsen
Preclinical, Trial termination: BAX 802 in CHA With Inhibitors (clinicaltrials.gov) - Feb 2, 2021
P3, N=14, Terminated,
In our report, the appearance of low-titre inhibitors against rpFVIII, was not found to be clinically significant. Suspended --> Terminated; Takeda has determined that the benefit/risk profile does not support continuation of the surgery Study for this specific Congenital Hemophilia A with Inhibitors patient population.
- |||||||||| Hemlibra (emicizumab-kxwh) / Roche
[VIRTUAL] Real-World Safety of Emicizumab: The First Interim Analysis of the European Haemophilia Safety Surveillance (EUHASS) Database (Poster Hall (Virtual Meeting)) - Nov 5, 2020 - Abstract #ASH2020ASH_3483;
However, early data from the first Phase III trial, HAVEN 1, identified a risk for thrombotic microangiopathy (TMA) or thrombotic events (TEs) when emicizumab was used alongside activated prothrombin complex concentrate (aPCC [FEIBA]; dosed on average a cumulative amount of >100 U/kg/24 hours for ≥24 hours) leading to a warning in the label and ongoing safety monitoring...Concurrent treatments included recombinant activated factor VII (rFVIIa; NovoSeven®; n=23 PwHA), factor VIII, (FVIII products other than Obizur®; n=9 PwHA) and aPCC (n=1 PwHA)...No new or emerging safety signals for emicizumab were identified. However, this analysis was limited by the low number of emicizumab treated PwHA—especially in those without FVIII inhibitors, and relatively short exposure time to emicizumab.
- |||||||||| [VIRTUAL] Implication of Approval UNDER Exceptional Circumstances on Reimbursement in France () - Oct 3, 2020 - Abstract #ISPOREU2020ISPOR-EU_482;
Most of these drugs (84,6%) had non-comparative trials and limited/lack of evidence on clinical efficacy benefit.CONCLUSIONSEven with limited clinical evidence, HAS issued positive recommendations for reimbursement of drugs approved under EC, allowing faster patient access to drugs with no available alternatives and high unmet need. In France, single-arm trial and limited efficacy data does not seem an obstacle for reimbursement of these promising drugs for untreated diseases.
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