Hemlibra's indications are expanded

Designated as the first 'Orphan drug' to prevent non-antibody severe hemophilia A

The only subcutaneous injection method in Korea, up to once every 4 weeks

As a global new drug, indications & dosage standards for Hemlibra, a hemophilia preventive drug, have been expanded.

 

JW Pharmaceutical said on the 31st, that Hemlibra (Emicizumab), which dramatically improves the convenience of administration, is additionally approved by the MFDS as a routine preventive therapy for non-antibody patients.

 

Hemlibra is a genetic recombination drug that is a routine preventive therapy for hemophilia A caused by the deficiency of factor VIII of blood clotting.

 

It is a first in class drug that bridges activated factor IX and factor X to restore the function of missing activated factor VIII that is needed for effective hemostasis.

 

Following the change in the permit, Hemlibra expanded the scope of treatment to severe A hemophilia patients who did not have antibodies within a year after being approved as a preventive treatment for hemophilia A with antibodies earlier this year.

 

Hemlibra is currently marketed in more than 90 countries, including the United States, Japan, and Germany, and is recognized worldwide for its drug effect as a first in class drug.

 

It has already been approved for antibody patients in over 90 countries, and has been approved for prescription in non-antibody patients in over 70 countries.

 

In addition, the indications and dosages are not limited to the existing once-weekly subcutaneous injection method, and the dosage interval can be increased at the discretion of the medical staff.

 

All of the prophylactic treatments released so far had to be intravenously administered 2-3 times a week, but Hemlibra can be administered subcutaneously once a week, once every two weeks, and up to once every 4 weeks, dramatically improving patient dosing convenience.

 

Helimbra is the only drug in Korea that has been approved for the prevention of hemophilia, which is administered directly subcutaneously (under the skin) rather than intravenously (intravascular).

 

Helimbra was also designated as an orphan drug for the first time in February, as a treatment for severe non-antibody hemophilia.

 

According to the MFDS' notice, △ diseases where the number of patients in Korea is 20,000 or less △diseases that do not have appropriate treatment methods or drugs, or only drugs with significantly improved safety or effectiveness than existing alternative drugs are designated as orphan drugs An official from JW Pharmaceutical said that the company has been continuously pushing for permission to improve the quality of life for people with hemophilia A who need treatment for a lifetime.

 

And, since most patients with hemophilia A do not have antibodies, he expects more patients will be able to benefit from treatment by expanding the indications.

 

The domestic hemophilia treatment market size is estimated to be about ₩150 billion (Source: the HIRA).

 

Hemophilia is classified according to the type of insufficient blood coagulation factor, and type A hemophilia caused by the lack of factor VIII of blood clotting accounts for more than 80% of all hemophilia.

 

As of 2018, there were a total of 1889 patients with hemophilia A in Korea, 51 patients (3%) with double antibody and 1838 patients (97%) with non-antibody.