JW Pharmaceutical said Wednesday that the Ministry of Food and Drug Safety (MFDS) has granted orphan drug designation to Roche’s Hemlibra (Ingredient: emicizumab-kxwh), a type A hemophilia treatment drug.
The company had previously secured exclusive domestic sales right for the drug from Chugai Pharmaceutical, an affiliate of Roche in May.
Hemlibra is an antibody that reduces the frequency of bleeding episodes in adults and children with hemophilia type A with factor VIII inhibitors. It is a first-in-class drug with a bispecific antibody, which is a technology that mimics the action mechanism of factor VIII and binds to activated factor IX and X simultaneously.
The drug had received approval from the U.S. Food and Drug Administration (FDA) in November of last year. During its biologics license application (BLA), the FDA designated the drug as a breakthrough therapy and orphan drug. Such designations allowed the treatment to receive approval three months ahead of schedule, compared to applying the medication through the Prescription Durg User Free Act (PDUFA).
The European Medicines Agency (EMA) also approved the medication in February of this year.
Hemlibra is the first hemophilia treatment approved by the FDA that injects the drug directly into the skin instead of intravenously.
Until now, patients had to intravenously inject other hemophilia treatment two or three times a week. However, for Hemlibra, patients only need to inject the therapy once a week subcutaneously. The drug also improved convenience as patients can insert the medication by themselves.
The company expects that the drug will become an optimal therapeutic agent for patients with factor VIII inhibitors as it is also effective for patients who have built resistant toward existing treatments.
“This is an innovative new drug that resolves the problems of existing therapeutic drugs,” a company official said. “The drug provides continuity of efficacy and convenience to not only patients with hemophilia type A, who have antibodies, but also to patients who do not possess such antibodies.”
Since the drug received orphan drug status the company plans to quickly finish the remaining approval process so that it can contribute to improving the quality of life of people with hemophilia type A, he added.