Chugai's HEMLIBRA® Receives Breakthrough Therapy Designation from U.S. FDA for Hemophilia A Without Factor VIII Inhibitors
Chugai Pharmaceutical Co., Ltd. (TOKYO:4519) announced today that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation for its anti-coagulation factor IXa/X humanized bispecific monoclonal antibody / coagulation factor VIII substitute, “HEMLIBRA®” [US generic name: emicizumab-kxwh] for people with hemophilia A without factor VIII inhibitors. Development and distribution of the drug in the US is conducted by Genentech, a member of Roche Group.
“We are thrilled that HEMLIBRA has been granted its second Breakthrough Therapy Designation,” said Chugai’s Executive Vice President, Co-Head of Project & Lifecycle Management Unit, Dr. Yasushi Ito. “This will allow us to expedite potential delivery of this new therapy we created to people with hemophilia A without inhibitors in the US following the previous designation for inhibitors. We continue to work closely with Genentech to enable this line extension as soon as possible.”
This designation is based on the global phase III HAVEN 3 (NCT02847637) study evaluating HEMLIBRA subcutaneous injection once a week and once every two weeks in people with hemophilia A (12 years of age or older) without inhibitors to factor VIII.
Hemophilia A is a disease presenting repeated severe bleeding symptoms. In this disease, the blood coagulation reaction does not proceed normally due to the deficiency or functional disorder of coagulation factor VIII. For people with hemophilia A without inhibitors, regular factor VIII replacement therapy has been widely used to prevent bleeding. HEMLIBRA is a bispecific monoclonal antibody, which was developed using Chugai’s proprietary antibody engineering technologies. The drug is designed to bind factor IXa and factor X. In doing so, HEMLIBRA provides the cofactor function of factor VIII in people with hemophilia A, who either lack or have impaired coagulation function of factor VIII1, 2). In the HAVEN 3 study, a statistically significant reduction in the frequency of bleeding episodes was observed with HEMLIBRA. With the convenience of subcutaneous administration and the lower frequency of administration, it is expected to be a new treatment option for hemophilia A.
This is the sixth Breakthrough Therapy Designation received for three drugs created by Chugai: ALECENSA® (ALK-positive non-small cell lung cancer with disease progression on crizotinib, and first line treatment for ALK-positive non-small cell lung cancer), ACTEMRA® (systemic sclerosis and giant cell arteritis), and HEMLIBRA (prophylactic treatment for patients 12 years or older with hemophilia A with factor VIII inhibitors).
Trademarks used or mentioned in this release are protected by law.
1. Kitazawa, et al. Nature Medicine 2012; 18(10): 1570
2. Sampei, et al. PLoS ONE 2013; 8: e57479
About Breakthrough Therapy
Breakthrough Therapy Designation was adopted as part of the FDA Safety and Innovation Act (FDASIA) enacted in July 2012 aiming at expediting the development and review of drugs for the treatment of severe or life-threatening diseases or symptoms. In order to grant Breakthrough Therapy Designation, preliminary clinical evidence is required demonstrating that the drug may have substantial improvement on at least one clinically significant endpoint over existing therapies. Breakthrough Therapy Designation includes the features of a Fast Track designation, with the addition of intensive guidance on efficient drug development as well organizational commitment from FDA.
Main approval status of the drug
US: In November 2017, the drug (US product name: HEMLIBRA®; Genentech) was approved by the U.S. Food and Drug Administration and was marketed for “routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adult and pediatric patients with hemophilia A (congenital factor VIII deficiency) with factor VIII inhibitors.”
EU: In Europe, the drug (EU product name: HEMLIBRA®; Roche) obtained regulatory approval from the European Commission and was marketed for routine prophylaxis of bleeding episodes in people with hemophilia A with factor VIII inhibitors in February 2018.
Japan: The Ministry of Health, Labour and Welfare has approved HEMLIBRA for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in patients with congenital factor VIII deficiency (hemophilia A) with factor VIII inhibitors in March 2018.
About the results of HAVEN 3 study
Press release issued on November 20, 2017
Chugai Pharmaceutical is one of Japan’s leading research-based pharmaceutical companies with strengths in biotechnology products. Chugai, based in Tokyo, specializes in prescription pharmaceuticals and is listed on the 1st section of the Tokyo Stock Exchange. As an important member of the Roche Group, Chugai is actively involved in R&D activities in Japan and abroad. Specifically, Chugai is working to develop innovative products which may satisfy the unmet medical needs, mainly focusing on the oncology area.
In Japan, Chugai’s research facilities in Gotemba and Kamakura are collaborating to develop new pharmaceuticals and laboratories in Ukima are conducting research for technology development for industrial production. Overseas, Chugai Pharmabody Research based in Singapore is engaged in research focusing on the generation of novel antibody drugs by utilizing Chugai’s proprietary innovative antibody engineering technologies. Chugai Pharma USA and Chugai Pharma Europe are engaged in clinical development activities in the United States and Europe.
The consolidated revenue in 2017 of Chugai totaled 534.2 billion yen and the operating income was 103.2 billion yen (IFRS Core basis).
Additional information is available on the internet at https://www.chugai-pharm.co.jp/english.
Chugai Pharmaceutical Co., Ltd.
Media Relations Group, Corporate Communications Dept.,
For US media
Chugai Pharma USA Inc.
For European media
Chugai Pharma France SAS
For Taiwanese media
Chugai Pharma Taiwan Ltd.
Chugai Pharmaceutical Co., Ltd.
Investor Relations Group, Corporate Communications Dept.,
Om Business Wire
(c) 2018 Business Wire, Inc., All rights reserved.
Business Wire, a Berkshire Hathaway company, is the global leader in multiplatform press release distribution.
Følg saker fra Business Wire
Registrer deg med din epostadresse under for å få de nyeste sakene fra Business Wire på epost fortløpende. Du kan melde deg av når som helst.
Siste saker fra Business Wire
myPOS Presented with Best POS Innovation Award by the Merchant Payment Ecosystem21.2.2019 14:57:00 | Pressemelding
myPOS, one of the fastest growing European payment providers was handed the Best POS Innovation/POS Software Payment Applications award for 2019 by the Merchant Payment Ecosystem (MPE) at a Gala Dinner ceremony in Berlin last night. Being known for offering instant settlement and no-monthly-fees model to SMEs across Europe, myPOS provides a range of innovative features on its payment devices, some of which include Tipping, Payment Request, Top-up and GiftCards. In addition, myPOS merchants are entitled to a whole range of subscription-free, value-added services such as Virtual MO/TO, PayLinks and PayButtons, Checkout module with the most popular shopping carts and more. myPOS clients are also given the opportunity to manage their funds with a free online account and a unique banking platform. The company got nominated in two categories: Best POS Innovation and Best Acquirer of the year, but ultimately won the Best POS Innovation award. "We accept this award with gratitude and appreciat
New Release Crea Records: "ABBA We Love You Forever"21.2.2019 14:44:00 | Pressemelding
Not many people on this earth can say that they have never heard of ABBA. In addition to the wonderful voices of Agnetha Fältskog and Anni-Fried Lyngstad, the musicians, Björn Ulvaeus and Benny Andersson have written the most wonderful songs, not only composed with charm and elegance, but also so breathtakingly beautiful and unforgettable that they will always resonate. For the Danish singer, BILLBOARD Hot 100 artist, composer and lyricist, Lecia Jonsson, ABBA has been much more than an inspiration. Lecia shared the same melodic universe as ABBA, making her mark with many records as part of duo LECIA & LUCIENNE. Later, as part of another duo, LABAN, Lecia entered BILLBOARD HOT 100 in the United States, had releases in 48 countries worldwide, and sold more than 1.5 million albums. Lecia’s identity is formed by her pure voice and her great sense for writing unique songs filled with a great melodic substance. If anyone should write a song to honour ABBA’s music, Lecia is definitely the pe
Ultivue Expands Global Presence with Opening of European Subsidiary, Ultivue EMEA Srl21.2.2019 14:30:00 | Pressemelding
Ultivue, a developer of tissue biomarker identification and quantification assays for pathology and translational research labs, today announced that it has expanded its commercial footprint with a wholly-owned European subsidiary and appointed Luigi Pirovano as General Manager. Mr. Pirovano is an international executive with significant experience managing European Diagnostics and Life Science operations and will be responsible for managing the new subsidiary, Ultivue EMEA Srl, located in Milan, Italy. “The establishment of a European subsidiary provides an excellent structure to support Ultivue’s expanded commercial activities across Europe and engage deeply and efficiently with the biomedical community,” said Philippe Mourere, Ultivue’s Senior Vice President of Commercial Operations. “Ultivue will capitalize on Luigi’s demonstrated success leading both Life Science and Diagnostics activities to continue driving strong execution of its business plan.” Luigi Pirovano has more than 30
Seoul Semiconductor Made Global Distributor Stop Selling Everlight Product in Japan21.2.2019 14:00:00 | Pressemelding
Seoul Semiconductor Co., Ltd. (KOSDAQ 046890) (“Seoul”), a leading global innovator of LED products and technology, announced that it has concluded a patent infringement lawsuit seeking an injunction on the sales of certain LED product sold by Mouser Electronics (“Mouser”). The accused LED product was manufactured by Everlight Electronics Co., Ltd. (“Everlight”). This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20190221005040/en/ Seoul Semiconductor's Headquarters in Korea (Photo: Business Wire) In February 2018, Seoul filed a patent infringement lawsuit with the Tokyo District Court, accusing the LED product manufactured by Everlight and sold by Mouser of infringing an LED patent of Seoul’s affiliate. Mouser agreed not to export the accused Everlight LED product in Japan, and Seoul therefore agreed to withdraw the lawsuit. The patented technology involved in this litigation serves to efficiently extract light emitted from the i
NTT DATA Launches Advanced 3D Digital Map Package for 5G Network Planning21.2.2019 14:00:00 | Pressemelding
NTT DATA (TOKYO:9613), a leading IT services provider, announced today its launch of “AW3D Telecom for 5G,” an advanced 3D digital map package that leverages high-quality satellite imagery for the planning of fifth-generation (5G) wireless networks, effective immediately. AW3D for 5G is expected to be used by telecom carriers, network vendors and IoT companies engaged in businesses involving 5G networks. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20190221005320/en/ Images of AW3D Telecom for 5G (Graphic: Business Wire) Commercial 5G services via fixed wireless access networks launched in the USA in 2018 and will begin operating in Japan, South Korea, the UK and Australia in 2019. Accurate 3D models are crucial for designing 5G networks, which use millimeter-spectrum waves that are highly sensitive to interference from natural and manmade objects. Buildings, trees, bridges, flyover roads, etc. need to be expressed precisely
Universal Laser Systems Maximizes Material Compatibility in New ULTRA 9 Platform21.2.2019 13:05:00 | Pressemelding
With the increasing use of advanced materials in industrial applications from aerospace to medical devices comes a growing need for innovation in material conversion technology. Developments in laser processing aim to overcome the limitations of existing technology and push the boundaries of how materials can be used. To this end, Universal Laser Systems has released a platform with unprecedented material processing capabilities. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20190221005340/en/ ULTRA 9 laser platform (Photo: Business Wire) The new ULTRA 9 Platform is designed to perform laser cutting, laser ablation, and laser surface modification. When configured with patented MultiWave Hybrid™ technology, it can combine the laser energy of up to three wavelengths – 9.3 µm (CO2), 10.6 µm (CO2), and 1.06 µm (fiber) – by independently controlling each spectral component of the beam. The user is able to select the ideal waveleng