Teijin Limited announced its agreement with UK pharmaceutical company Sigma-Tau Pharma Ltd Opening a new window., part of the Sigma-Tau Group, for exclusive development and distribution rights in Japan to EZN-2279, a therapeutic agent for adenosine deaminase (ADA) deficiency in patients with severe combined immune deficiency (SCID).

EZN-2279, an injectable recombinant ADA, suppresses a reduction in the number of lymphocytes by replacing ADA. Sigma-Tau Pharma is currently engaged in Phase 3 development of EZN-2279 in the United States.

Hiroshi Uno, president of Teijin Pharma Limited Opening a new window, the core company of the Teijin Group's medical and pharmaceutical business, said: “Teijin Pharma is scheduled to commence Phase 3 clinical trials in Japan within the current fiscal year ending in March 2015. Ultimately, we hope to contribute to the quality of life for patients with ADA deficiency by providing them with the option of Japan’s first ADA enzyme replacement therapy.”

EZN-2279 is one of the unapproved drugs with high medical needs that have been publicly offered for development by the Review Committee on Unapproved or Off-Label Drugs with High Medical Needs under the Ministry of Health, Labour and Welfare to encourage the development of unapproved or off-label drugs by pharmaceutical companies. Teijin Pharma is the successful applicant for EZN-2279.

ADA deficiency is a disorder caused by a lack of the enzyme adenosine deaminase due to a gene mutation. This leads to a decreased number of lymphocytes, increased susceptibility to infectious diseases and possibly SCID and growth impairments. ADA deficiency, which affects one in 200,000 to 1,000,000 persons, is designated as an intractable disease eligible for government funding in Japan.

The first treatment option for ADA deficiency is hematopoietic stem cell transplantation. In cases where transplantation is difficult, doctors may adopt either enzyme replacement therapy by injecting ADA, which has yet to be approved in Japan, or gene therapy.

Sigma-Tau Pharma Ltd, is part of the Sigma-Tau Group, a privately owned company, based in Pomezia, Italy. Sigma-Tau Pharma’s mission is to develop and make available to patients and Health Care Professionals innovative products for the treatment of Rare Orphan Diseases. The company is based in Windsor (UK), has R&D facilities in Switzerland and the USA and markets its products through affiliates and partners in over 60 countries.

Teijin (TSE: 3401) is a technology-driven global group offering advanced solutions in the areas of sustainable transportation, information and electronics, safety and protection, environment and energy, and healthcare. Its main fields of operation are high-performance fibers such as aramid, carbon fibers & composites, healthcare, films, resin & plastic processing, polyester fibers, products converting and IT. The group has some 150 companies and around 16,000 employees spread out over 20 countries worldwide. It posted consolidated sales of JPY784.4 billion (USD 7.7 billion) and total assets of JPY 768.4 billion (USD 7.5 billion) in the fiscal year ending March 31, 2014.