European Commission approves Roche ’s Hemlibra for people with severe haemophilia A without factor VIII inhibitors

Roche today announced that the European Commission has approved Hemlibra ® (emicizumab) for routine prophylaxis of bleeding episodes in people with severe haemophilia A (congenital factor VIII deficiency, FVIII
Source: Roche Investor Update - Category: Pharmaceuticals Source Type: news

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Basel, 29 June 2020 - Roche (SIX: RO, ROG; OTCQX: RHHBY) today announced that new data from its haemophilia A clinical programme will be presented at the International Society on Thrombosis and Haemostasis (ISTH) 2020 Congress on 12-14 July 2020. Data will include updated safety results from the phase IIIb STASEY study of Hemlibra ® (emicizumab) and new results from the phase III HAVEN 5 study of Hemlibra. Data will also include insights into the impact of living with haemophilia A. Spark Therapeutics (a member of the Roche Group) will also present data from the initial dose cohorts of its phase I/II SPK-8011 gene ther...
Source: Roche Media News - Category: Pharmaceuticals Source Type: news
Acquired Haemophilia A is a rare acquired bleeding disorder caused by autoantibodies directed against Factor VIII, which neutralize FVIII activity. These inhibitors differ from alloantibodies against FVIII which can occur in congenital Haemophilia A after repeated exposures to plasma-derived or recombinant FVIII products. In most cases the disease occurs suddenly in subjects without personal or familiar history of bleedings, with symptoms that may be mild, moderate or severe. However, only laboratory alterations are present in   ̴ 30% of patients. The incidence varies from 1 to 4 cases per million/year; more tha...
Source: Mediterranean Journal of Hematology and Infectious Diseases - Category: Hematology Authors: Source Type: research
Authors: Arain A, Muhsen IN, Abdelrahim M Abstract Acquired haemophilia is a severe haematological disorder characterised by the presence of anti-factor VIII antibodies. Although rare, it can lead to serious bleeding complications. Acquired haemophilia can be seen in patients with malignancies as a paraneoplastic phenomenon. This is a case of a 52-year-old patient who presented with haematuria and retroperitoneal bleeding soon after being diagnosed with pancreatic adenocarcinoma and subsequently was found to have acquired haemophilia. The treatment of underlying malignancy with chemotherapy may accelerate the eradi...
Source: Ecancermedicalscience - Category: Cancer & Oncology Tags: Ecancermedicalscience Source Type: research
Authors: Rodriguez-Merchan EC, De la Corte-Rodriguez H Abstract INTRODUCTION: Iliopsoas muscle hemorrhage in patients with hemophilia is a serious complication with relevant related morbidity. AREAS COVERED: A Cochrane Library and PubMed (MEDLINE) search of studies related to iliopsoas hematomas in hemophilia was analyzed. Pain in the hip joint area and femoral nerve involvement in people with hemophilia should alert physicians to the likelihood of a hemorrhage of the iliopsoas muscle. Clinically, reduced hip range of motion and deficient motor and sensory function of the femoral nerve can be found. Imaging stu...
Source: Expert Review of Hematology - Category: Hematology Tags: Expert Rev Hematol Source Type: research
Authors: Valentino LA, Khair K Abstract INTRODUCTION: Hemophilia A is a bleeding disorder traditionally managed with standard half-life (SHL) factor (F) VIII concentrates. Extended half-life (EHL) FVIII products and emicizumab-kywh, a nonfactor therapy, are newer treatment options. Additional nonfactor agents and gene therapy are expected to reach the market in the near future. AREAS COVERED: A PubMed (MEDLINE) search from 1962 to April 2020 related to hemophilia A, its management, and the products currently available for prophylaxis was performed to comprehensively review these topics and analyze the benefits ...
Source: Expert Review of Hematology - Category: Hematology Tags: Expert Rev Hematol Source Type: research
In conclusion, this study is designed to support haemophilia physicians using Emicizumab in physicians treating hemophilia and using (PWHAs). With further post-marketing experience and trials, regular updates are necessary. PMID: 32588417 [PubMed - as supplied by publisher]
Source: Hamostaseologie - Category: Hematology Authors: Tags: Hamostaseologie Source Type: research
et) Abstract INTRODUCTION:  Limited data exist on the clinical impact of low-responding inhibitors and the requirement for immune tolerance induction (ITI) treatment to establish tolerance, reduce bleeding, and improve outcome. The aim of this article is to describe the therapeutic management of children with severe hemophilia A and low-responding inhibitors and its effect on bleeding phenotype. METHODS:  The REMAIN (Real-life Management of Inhibitors) study is a satellite study of the PedNet registry. It included unselected children with severe hemophilia A (factor VIII [FVIII] 
Source: Thrombosis and Haemostasis - Category: Hematology Authors: Tags: Thromb Haemost Source Type: research
CONCLUSION: Data from the completed pathfinder2 trial, one of the largest and longest-running clinical trials to investigate treatment of severe hemophilia A, demonstrate the efficacy and safety of N8-GP in previously treated adolescent and adult patients. PMID: 32544297 [PubMed - as supplied by publisher]
Source: Thrombosis and Haemostasis - Category: Hematology Authors: Tags: J Thromb Haemost Source Type: research
In this report, we describe a patient with severe hemophilia A with inhibitors who developed a neutralizing anti-drug antibody to emicizumab, for whom we performed extensive testing in the special coagulation laboratory. PMID: 32544268 [PubMed - as supplied by publisher]
Source: Thrombosis and Haemostasis - Category: Hematology Authors: Tags: J Thromb Haemost Source Type: research
In conclusion, HbA reduces severity of HbS in HbS-β + thalassemia. HbS-HbD dis ease can manifest as a transfusion dependent illness. HbSS reduces severity of G6PD deficiency after first few years of life. HbSS and hemophilia coinheritance ameliorates symptoms of hemophilia.
Source: Indian Journal of Hematology and Blood Transfusion - Category: Hematology Source Type: research
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