— The Fifth Breakthrough Therapy Designation Granted for a Chugai
Originated Drug —
Pharmaceutical Co., Ltd. (TOKYO:4519) announced that the US Food and
Drug Administration (FDA) has granted Breakthrough Therapy Designation
to ACTEMRA®/RoACTEMRA® (tocilizumab), a Chugai
originated drug, which is currently under development by Roche and
Genentech for the indication of Giant Cell Arteritis (GCA).
“We are very pleased that the FDA has once again granted Breakthrough
Therapy Designation to ACTEMRA/RoACTEMRA, following last year’s
designation for systemic sclerosis,” said Chugai’s Senior Vice
President, Head of Project & Lifecycle Management Unit, Dr. Yasushi Ito.
“This designation indicates that ACTEMRA/RoACTEMRA is highly regarded,
and has great potential to fulfill unmet medical needs in auto-immune
This designation was based on the GiACTA study, which is a global Phase
III study assessing the efficacy and safety in patients with GCA. This
is the fifth Breakthrough Therapy Designation for a Chugai originated
drug, following these three products: alectinib (ALK-positive non-small
cell lung cancer with disease progression on crizotinib, first line
treatment for ALK-positive non-small cell lung cancer), tocilizumab
(systemic sclerosis), and emicizumab (prophylactic treatment for
patients 12 years or older with hemophilia A with factor VIII
Based on Chugai’s business philosophy “innovation all for the patients,”
Chugai will collaborate with Roche and Genentech to submit marketing
applications for ACTEMRA/RoACTEMRA in a number of countries around the
world, with the intent to increase access to this new treatment option
for patients and healthcare professionals as soon as possible.
About Breakthrough Therapy
The 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.
About Giant Cell Arteritis
Giant Cell Arteritis (GCA)
belongs to an autoimmune disease called large-vessel vasculitis. GCA is
a granulomatous vasculitis occurring primarily in the aorta and aortic
branches, mainly the temporal arteries. Common initial symptoms include
headache, systemic conditions such as fever, and loss of vision. GCA is
prevalent in Western countries and affects women more than men with the
typical age of onset 50 years or older1). Vasculitis can be
classified into three different groups depending on the size of the
inflammatory vessels, such as large vessel vasculitis, medium vessel
vasculitis and small vessel vasculitis2). Besides GCA,
Takayasu’s arteritis which appears more commonly in Asian young ladies
is also included in large-vessel vasculitis.
About the GiACTA Study
GiACTA (NCT01791153) is a Phase III,
global, randomised, double-blind, placebo-controlled trial investigating
the efficacy and safety of ACTEMRA®/RoACTEMRA® as
a novel treatment for GCA. It is the largest clinical trial ever
conducted in GCA and the first to use blinded, variable-dose,
variable-duration steroid regimens. The multicenter study was conducted
in 251 patients across 76 sites in 14 countries. The study’s primary
endpoint was the proportion of patients achieving sustained disease
remission at week 52. The secondary endpoints were the time to first GCA
flare after clinical remission, cumulative corticosteroid dose at week
52, and also safety outcome measures.
The GiACTA data will be
submitted for presentation at an upcoming medical conference and to
regulatory authorities around the world for approval consideration.
1. Lawrence C, et al. Arthritis & Rheum 1998; 41: 778-99
Jennet JC, et al. Arthritis & Rheum 2013; 65: 1-11
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.
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 2015 of
Chugai totaled 498.8 billion yen and the operating income was 90.7
billion yen (IFRS Core basis).
Additional information is available
on the internet at http://www.chugai-pharm.co.jp/english.
Chugai Pharmaceutical Co.,
Media Relations Group, Corporate Communications Dept.,
Chugai Pharma USA Inc.
Chugai Pharma France SAS
Chugai Pharma Taiwan Ltd.
Chugai Pharmaceutical Co., Ltd.
Relations Group, Corporate Communications Dept.,