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CSL Receives Manufacturing and Marketing Authorization in Japan for “Berinert® S.C. Injection 2000,” as a Medicine for prevention of Acute Hereditary Angioedema (HAE) attacks

KING OF PRUSSIA, PA

At-home subcutaneous therapy advances treatment options for patients and marks a milestone in 40 years of delivering on CSL’s promise to the HAE patient Community

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CSL Behring K.K. (Head Office: Minato-ku, Tokyo; President and Representative Director: Jean-Marc Morange) announces that it has received a manufacturing and marketing approval from the Ministry of Health, Labour and Welfare for “Berinert® S.C. Injection 2000,” a lyophilized human C1-esterase inhibitor concentrate for subcutaneous (SC) injection in plasma derivative, for the prevention of acute hereditary angioedema (HAE) attacks. “Berinert® S.C. Injection 2000” is a twice-weekly subcutaneous injection formulation of Berinert 60 IU/kg that can be self-administered at home.

“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,” said Haruo Kitado, Head of R&D Japan. 

The approval of "Berinert® S.C. Injection 2000" was based on the evaluation of the efficacy and safety from the global Phase III COMPACT (Clinical Study for Optimal Management of Preventing Angioedema with low-volume subcutaneous C1-inhibitor replacement Therapy) Study,1 the COMPACT long-term administration study (Open-label Extension),2 and a Japanese Phase III clinical study. In the COMPACT study, acute HAE attacks were significantly lower in the "Berinert" 60 IU/kg group at 0.52 per month compared to 4.03 per month in the placebo group (p<0.001, mixed model). In the domestic Phase III clinical study, the efficacy and safety of Berinert 60 IU/kg were confirmed to be similar in Japanese HAE patients as in the global Phase III study population, “These data reflect the potential of Berinert to help patients with HAE in Japan and underscore the value of Berinert as a prophylactic treatment for this population,” said Catherine Milch, Vice President R&D Immunology, CSL.

Jean-Marc Morange, President and Representative Director of CSL Behring K.K. states, “The human C1-esterase inhibitor preparation replaces lacking C1-INH in HAE patients. 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. As a pioneer in HAE treatment and prevention with more than 40 years of presence, we at CSL Behring are committed to making a difference in the lives of such patients by providing "Berinert® S.C. Injection 2000”, and to significantly advancing diagnosis and appropriate long term prophylaxis treatment to HAE patients in Japan.”

About HAE 
HAE is a rare, genetic and potentially life-threatening condition that causes painful, debilitating and unpredictable episodes of swelling of the abdomen, larynx, face and extremities, among other areas of the body. It is recognized as one of the diseases of "Primary Immunodeficiency Syndrome," a government-designated intractable disease.

It is reported that there are approximately 300 HAE patients diagnosed and undergoing treatment in Japan3. According to global data, there is one patient for every 50,000 people in the population4, and it is estimated that there are approximately 2,500 patients in Japan.5

About CSL Behring’s HAE community support in Japan
In June of this year in Japan, CSL Behring renewed its HAE Information Center, a disease awareness website, to provide information to HAE patients and their families. This renewal is intended to provide accurate information about the disease in an easy-to-understand manner, to alleviate the concerns and anxiety of patients and their families, and to contribute to improving the quality of life for patients.

In addition, CSL Behring has launched a "Tsunagu Project" under the slogan of "continuing to contribute to HAE patients with the utmost trust, both in the past and in the future". This is a company-wide effort to realize better HAE medical care by promoting closer partnership between CSL and medical professionals as well as between medical professionals and patients. The main goals of this initiative are to continue to listen to the voices of HAE patients and their families, bring forward innovations that address unmet needs. CSL Behring continues to improve the quality of life of patients with rare and intractable diseases including HAE patients and contribute to a better understanding of patients.

About Berinert® S.C
“Berinert® S.C. Injection 2000” has been marketed worldwide under the trade names of Berinert® or Haegarda®, in more than 14 countries and regions including the US, Europe, and Australia since 2017.

In Japan, "Berinert® P I.V. Injection 500," an intravenous formulation with the same active ingredient as "Berinert® S.C. Injection 2000," was approved and marketed in 1990 for the treatment of acute attacks of HAE, and in 2017 for the additional indication of prevention of acute attacks of HAE due to invasive procedures. “Berinert® S.C. Injection 2000” was developed as a subcutaneous injection formulation for the prevention of acute attacks of HAE. The indication of "Berinert® S.C. Injection 2000" was designated as an Orphan Drug on August 17, 2020, with the expected indication of "prevention of hereditary angioedema attacks".

Basic Information of “Berinert® S.C. Injection 2000”

Trade name

Berinert® S.C. Injection 2000

Indications or effects

Prevention of acute hereditary angioedema (HAE) attacks

Dosage and administration

Reconstitute this drug with the total volume of accompanying diluent and administer subcutaneously.

In general, administer 60 IU/kg twice a week.

Date of approval

September 26, 2022

Manufacturing and marketing

CSL Behring K.K.

 

About CSL
CSL (ASX:CSL; USOTC:CSLLY) is a leading global biotechnology company with a dynamic portfolio of lifesaving medicines, including those that treat haemophilia and immune deficiencies, vaccines to prevent influenza, and therapies in iron deficiency, dialysis and nephrology. Since our start in 1916, we have been driven by our promise to save lives using the latest technologies. Today, CSL – including our three businesses, CSL Behring, CSL Seqirus and CSL Vifor – provides lifesaving products to patients in more than 100 countries and employs 30,000 people. Our unique combination of commercial strength, R&D focus and operational excellence enables us to identify, develop and deliver innovations so our patients can live life to the fullest. For inspiring stories about the promise of biotechnology, visit CSLBehring.com/Vita and follow us on Twitter.com/CSL.
For more information about CSL, visit www.CSL.com.

Reference:

  1. Prevention of hereditary angioedema attacks with a subcutaneous C1 inhibitor. Longhurst H, et al. N Eng J Med. 2017;376:1131-1140.
  2. Long-Term Outcomes with Subcutaneous C1-Inhibitor Replacement Therapy for Prevention of Hereditary Angioedema Attacks. Craig et al. J Allergy Clin Immunol Pract. 7(6):1793–1802.
  3. Osawa I. Hereditary Angioedema. Iyaku Journal
  4. Longhurst HJ, Bork K. Hereditary angioedema: causes, manifestations, and treatment. Br J Hosp Med. 2006;67(12):654-657.)
  5. Horiuchi T. Hereditary Angioedema from 1888 to 2018 -Progress and Problems. Intern Med. 2018 Nov 1;57(21):3065-3066.


Media Contact
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