Japan gov't panel approves 2 iPS-derived drugs in global first

Japan's health ministry panel on Thursday approved the commercialization of two regenerative medicine products prepared from iPS cells, the first of their kind in the world.

The two drugs, ReHeart developed by Cuorips Inc., a startup originating from the University of Osaka, and Amchepry by Sumitomo Pharma Co. and Racthera Inc., will be used for patients with severe heart failure stemming from ischemic cardiomyopathy and Parkinson's disease, respectively.

"I am very happy to see the first big step toward its societal implementation, 20 years since it was announced," said Shinya Yamanaka, director emeritus at Kyoto University's Center for iPS Cell Research and Application.

Induced pluripotent stem cells, or iPS, were generated by Yamanaka, who announced the generation of mouse iPS cells in 2006 and human iPS cells in 2007. He won the Nobel Prize in Physiology or Medicine in 2012.

With ReHeart, heart muscle sheets derived from human iPS cells are placed on the surface of the heart, where they promote the formation of new blood vessels underneath and help restore heart function.

Patients with severe heart disease are often given medicine to prevent deterioration and sometimes may have to undergo a heart transplant.

In a clinical trial, the heart muscle sheets were transplanted into eight patients. The trial showed reduced symptoms, such as exhaustion and heart palpitations, as well as improvements in heart function and physical fitness.

With Amchepry, human iPS cells are grown into "dopaminergic neural progenitor cells" and injected into the patient's brain.

The cells were transplanted into seven patients in a clinical trial. Of the six assessed for efficacy, the transplanted cells produced dopamine, and some patients also showed improvement in motor symptoms.

Parkinson's disease is a progressive neurodegenerative disease caused by a degeneration and loss of dopamine-producing brain cells, which can lead to a decrease in motor function.

The panel set an approval term of seven years, with the product set to roll out after discussions regarding price, insurance coverage and preparations for manufacturing and distribution.

The products were approved in a limited capacity as the clinical studies for both products were conducted on a small number of patients. If they are later deemed safe, the products can be fully utilized without restrictions.

Cuorips said it aims to start selling its product within this year.

© KYODO