Amicus Therapeutics K.K.

The origin of Amicus dates to when the two children of John Crowley, Chairman and CEO, developed Pompe disease with a life expectancy of only 5 years. To save his children’s life, Crowley became President & CEO of a biotechnology company that was researching a treatment for Pompe disease. After many twists and turns, John’s children were able to participate in the clinical study of an investigational drug. As a result, his two children survived and have now reached adulthood. In 2002, John assumed an executive role at Amicus, later becoming Chairman and CEO of the company. The Hollywood movie Extraordinary Measures, released in 2010, is based on the story of the Crowley family and encourages patients and families with intractable diseases around the world. In Japan, Fuji TV reported the story as part of its popular program Unbelievable in July 2010, making the family’s story, and Pompe disease, widely known in this country as well.

In addition to Galafold®, AT-GAA, an investigational therapy for the treatment of Pompe disease, is currently in late stage development. Pompe disease is a genetic disorder in which glycogen (a complex sugar) builds up in the body's cells. This buildup occurs because there is a deficiency of the acid alfa glucosidase (GAA) enzyme, which is responsible for breaking down glycogen in the body.

Ever committed to bettering the lives of those with life-threatening rare and orphan diseases, Amicus has a strong pipeline for additional treatments for other diseases including Batten disease, Mucopolysaccharidosis, and more.

Mr. Chris Hartz (General Manager, Amicus Therapeutics K.K.) says: “We believe our success is measured by the number of lives we can impact. For that we encourage and embrace constant innovation.”

In 2016, Amicus established its Japanese subsidiary in Tokyo and has more recently expanded to a larger office.

“Establishing an entity in Japan without any local partners was a huge challenge for a young company like us. When the Japan entity was formed, our first product Galafold® had only been approved in the EU 6 months previously. However, Amicus believed direct entry was the best way to bring our product, along with our unique corporate culture, to patients in Japan,” commented Chris Hartz (General Manager, Amicus Therapeutics K.K.).

The origin of chaperone therapy is as a result of the research by a Japanese doctor, Yoshiyuki Suzuki and Mr. Hartz went on to comment: “We are very proud that Japanese Fabry patients now receive the benefits of Dr. Suzuki’s initial research. We also hope to have many future opportunities to deliver new medicines for people living with rare metabolic diseases in Japan.”

The deal has provided Amicus with the largest portfolio of gene-therapy products for rare diseases in development, of any company in the world.

For the establishment of Amicus’ Japanese subsidiary, the JETRO Invest Japan Business Support Center (IBSC) offered consultation on market and regulatory matters, referred Amicus to service providers including bilingual recruitment agencies which specialize in the medical field, and provided market information.

Regarding JETRO’s support, Mr. Chris Hartz said, “JETRO provided such a high level of information and the requirements needed to set up the local entity. JETRO’s support saved time and resources, which was imperative for a start-up company like us who needed to act fast.”

（Interviewed October 2019）