Emicizumab, a new antibody treatment for hemophilia A with inhibitors, has been designated as an orphan drug in South Korea, raising hopes that Korean patients with the disease would soon benefit from significantly less treatment administration burden.

According to JW Pharmaceutical Corp., which has exclusive domestic marketing rights for emicizumab in Korea, on Wednesday, the Ministry of Food and Drug Safety has designated emicizumab, which was developed by Japan’s Chugai Pharmaceutical Co., a subsidiary of Switzerland-based F. Hoffman-La Roche Ltd., as an orphan drug.

The ministry designates orphan drugs to promote pharmaceutical companies’ research and development on rare diseases and expand treatment opportunities for patients. Its latest decision to designate emicizumab as an orphan drug has placed the drug on a fast-track approval process for its sale in Korea.

Hemophilia is a rare bleeding disorder that results from lack of clotting factors A or B. About 80 percent of people with hemophilia have reduced levels of clotting factor A or VIII.

Currently, existing hemophilia treatments require two to three intravenous infusions per week, but emicizumab can be administered by patients with a once-a-week injection beneath their skin, which is significantly less burden for the patients with hemophilia A with inhibitors.

The sale of the drug has been already approved by the U.S. Food and Drug Administration (FDA) in November last year and by the European Medicines Agency in February this year. It was the first time for a subcutaneous injection treatment for hemophilia A with inhibitors to be approved in the United States.

An unnamed official from JW Pharmaceutical said that now that emicizumab has been designated as an orphan drug, it will focus on speeding up the remaining approval process for the sale of the drug in Korea. The Korean company secured exclusive domestic marketing rights for emicizumab from Chugai Pharmaceutical last year.

Shares of JW Pharmaceutical ended at 43,850 won ($40.8), up 2.1 percent or 900 won from the previous session.

By Kim Hye-soon and Lee Eun-joo

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