Takeda's hereditary angioedema (HAE) treatment Takhzyro (lanadelumab) has expanded its indication to children aged two and older in the United States. However, few know when the drug will be released in Korea, although two years have passed since it won approval.
Takeda said the U.S. FDA approved the company’s additional biologics license application (BLA) for Takhzyro treating HAE attacks in children aged two to six on Feb. 4.
With the approval, patients aged two and older can be prescribed Takhzyro to prevent HAE.
As the age range is wide, there is a difference in usage by age. Patients aged two to six are given a 150 mg solution once every four weeks in a single-dose pre-filled syringe. Patients aged six to 12 are given the same dose every two weeks. For adolescents aged 12 or older and adults, a 300mg solution is administered every two weeks. Those remaining stable without seizures for more than six months can reduce administration to 300mg once every four weeks.
Angioedema is an allergic disease in which body fluid escapes from the blood vessels and the tissues swell. In HAE, only vascular edema appears without allergic reaction in the shallow layer of the skin's epidermis.
HAE attacks occur in various body parts, such as the abdomen, face, hands, and feet. If upper airway vascular edema occurs, it is fatal to the patient.
According to the SPRING study, an open-label phase 3 clinical trial on 21 HAE patients aged two to 12, the Takhzyro group reduced the HAE attack rate by 94.8 percent on average compared to the standard treatment during 52 weeks of treatment, from 1.84 to 0.08 case a month, with 16 (76.2 percent) subjects showing the disease-free status of 99.5 percent on average.
“However, these results came from an open, uncontrolled test,” Takeda said. “Further corroborative studies are needed to draw statistical data conclusions.”
Meanwhile, Takhzyro received approval from the Ministry of Food and Drug Safety in February 2021 for adolescents aged 12 or older and adults. However, its market release has not been made until now.
Youngpoong Pharmaceutical’s male hormone treatment Danazol is currently used to prevent HAE attacks. However, women have trouble using it due to side effects. Takeda's Firazyr (icatibant acetate) is also used as a treatment for HAE attacks. However, the need to introduce preventive therapy is increasing because the drug gets insurance coverage for just two prescriptions and causes recurrence.
Korean patients who need Takhzyro can use it through the Korea Orphan & Essential Drug Center (KOEDC). Still, it takes lots of work, considering the procedures and cost, according to a patient group.
"Takeda Korea is trying its best to help Korean patients use Takhzyro to prevent HAE attacks,” a company official said.