Chugai’s HEMLIBRA® Receives Breakthrough Therapy Designation from U.S. FDA for Hemophilia A Without Factor VIII Inhibitors

17-Apr-2018 Intellasia | BusinessWire | 12:00 PM Print This Post

-The Sixth Designation for Chugai Originated Drug-

TOKYO–(BUSINESS WIRE)–#biotechChugai
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):
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

About Chugai
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


For Media
Chugai Pharmaceutical Co.,
Media Relations Group, Corporate Communications Dept.,
Tel: +81-3-3273-0881
E-mail: [email protected]
US media

Chugai Pharma USA Inc.
Casey Astringer
E-mail: [email protected]
European media

Chugai Pharma France SAS
Nathalie Leroy
E-mail: [email protected]
Taiwanese media

Chugai Pharma Taiwan Ltd.
Susan Chou
E-mail: [email protected]

Chugai Pharmaceutical Co., Ltd.
Relations Group, Corporate Communications Dept.,
Toshiya Sasai
E-mail: [email protected]


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