Japan : Anti-Coagulation Factor IXa/X Humanized Bispecific Monoclonal Antibody HEMLIBRA Subcutaneous Injection Approved in Japan.
With this approval, Im very thrilled that the day that HEMLIBRA will be available to people with hemophilia A in Japan is approaching, said Chugais President and CEO, Tatsuro Kosaka. HEMLIBRA is a first-in-class antibody developed with Chugais proprietary antibody engineering technologies, aiming to tackle unmet medical needs. We believe that the drug will bring innovation to people with hemophilia A and their family with its clinical aspects as well as by allowing a once-weekly subcutaneous injection.
This approval is based on two pivotal clinical studies for people with hemophilia A with inhibitors: the results of HAVEN 1 study (NCT02622321) for adolescents and adults, and the interim analysis of HAVEN 2 study (NCT02795767) in children.
HEMLIBRA is a bispecific monoclonal antibody, which was developed using Chugais proprietary antibody engineering technologies. The drug is designed to bind factor IXa and factor X. In doing so, HEMLIBRA provides the cofactor function of factor VIII in people with hemophilia A, who either lack or have impaired coagulation function of factor VIII1, 2). In November 2017, the drug (US product name: HEMLIBRA; Genentech) was approved by the U.S. Food and Drug Administration and was marketed 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. In Europe, it obtained regulatory approval from the European Commission for routine prophylaxis of bleeding episodes in people with hemophilia A with factor VIII inhibitors in February 2018 (EU product name: HEMLIBRA; Roche).
Hemophilia is an inherited, serious bleeding disorder where a persons blood does not clot properly, leading to uncontrolled bleeding which can occur spontaneously. Hemophilia consists of hemophilia A and hemophilia B, caused by the lack of factor VIII and factor IX, respectively. In Japan, it is reported that about 5,000 people are hemophilia A and about 1,000 people are hemophilia B3). The current standard treatment for hemophilia A is factor VIII replacement therapy and 25-30% of people with severe hemophilia A develop inhibitors to factor VIII replacement therapies4). Inhibitors are antibodies that attack and destroy the replaced factor VIII, because it is recognized as a foreign substance. Once people acquire inhibitors, it is expected to be difficult for them to administer factor VIII replacement therapies.
Conditions for approval
A risk management plan should be created and appropriately implemented.
Because the number of participants in Japanese clinical trials was very limited, post-marketing drug use surveillance of all patients receiving HEMLIBRA treatment should be conducted until data for a certain number of patients have been accumulated, in order to understand background information on people using HEMLIBRA as well as to collect safety and efficacy data on HEMLIBRA promptly, and take necessary measures for the appropriate use of HEMLIBRA.
Early phase post-marketing vigilance should be conducted.
All-case registration surveillance
The all-case registration surveillance is scheduled to collect the data of approximately 100 people who receive HEMLIBRA. The data will be reviewed to determine whether a new surveillance or further safety measures should be conducted. Results of the surveillance will be reported to the regulatory authorities, and the data shall be announced at future scientific meetings.
[c] 2018 Al Bawaba (Albawaba.com) Provided by SyndiGate Media Inc. ( Syndigate.info ).
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|Date:||Mar 24, 2018|
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