Japan OK’s Berinert SC Injection 2000 to Prevent HAE Attacks
The C1-inhibitor replacement therapy is designed to be self-administered
Japan’s Ministry of Health, Labour and Welfare has approved Berinert S.C. Injection 2000 — a C1-inhibitor replacement therapy designed to be self-administered at home via injection under the skin twice per week — for preventing swelling attacks in people with hereditary angioedema (HAE).
The therapy has been approved in the U.S., Australia, and Europe since 2017, and marketed under the brand names Berinert and Haegarda. In Japan, Berinert P I.V. Injection 500, an into-the-vein formulation with the same active ingredient as Berinert S.C. Injection 2000, has been available since the 1990s for treating acute HAE attacks.
The approval was announced by CSL Behring, which manufactures the treatment.
“We believe that the approval of ‘Berinert S.C. Injection 2000,’ a human C1-inhibitor preparation, for long term prophylaxis indication that can be self-administered at home, is a new and promising treatment option for patients,” Jean-Marc Morange, president and representative director of CSL Behring K.K., said in a press release.
“This regulatory milestone underscores CSL’s ongoing promise and investment in the health and well-being of the people in Japan – and the value of these innovative treatments,” Haruo Kitado, the company’s head of research and development in Japan, said.
According to CSL Behring, there are about 300 people in Japan diagnosed with HAE who are receiving treatment. Based on its typical prevalence, HAE is estimated to affect more than 2,000 people in the country.
The company recently renewed its HAE Information Center, a disease awareness website that provides information to people with HAE and their families in Japan. The company also has launched an initiative called “Tsunagu Project,” to promote partnerships between medical providers, patients, and CSL Behring in order to help those affected by HAE.
HAE types 1 and 2 are caused by mutations in the SERPING1 gene, which provides instructions for making a protein called C1-inhibitor. A dysfunctional C1-inhibitor leads to the uncontrolled production of a signaling molecule called bradykinin, which triggers swelling.
Berinert is a formulation of C1-inhibitor derived from plasma, the noncellular portion of blood, first approved in Germany in the late 1970s.
The new approval was supported by data from a Phase 3 trial called COMPACT (NCT01912456), which compared subcutaneous (under-the-skin) Berinert against a placebo in 90 people with HAE. Results showed Berinert, at a dosage of 60 international units per kilogram of body weight, significantly reduced the average number of swelling attacks compared with a placebo (0.52 vs. 4.03 attacks per month).
The approval was also supported by data from a Phase 3 study in Japan, which showed similar outcomes regarding the therapy’s effectiveness and safety profile. The most common side effects associated with subcutaneous Berinert in the COMPACT trial were injection site reactions, nearly all of which were mild and resolved shortly after onset.