From Jiji Press
February 26th 2020
TOKYO – A Japanese health ministry panel approved Wednesday the production and sales of a gene therapy for treating spinal muscular atrophy, a rare incurable disease that destroys muscle function through a decrease in motor neuron cells.
The health minister is expected to formally approve the therapy, Zolgensma, by the end of March, with public health insurance coverage seen starting this summer. The therapy, which has already been approved in the United States, is expensive, costing the dollar equivalent of 230 million yen in the country for one-time infusion.
At this time, the one-time-only gene therapy, developed by Swiss drug giant Novartis AG, will cover patients below two years old with abnormalities in their genes to make proteins necessary for maintaining motor nerves. It may be used for older patients as well in the future, informed sources said.
There are about 1,000 SMA patients in Japan. Fifteen to 20 people are believed to develop the disease annually in the country.
In the therapy, the proteins will be created after the genes carried by adeno-associated viruses, which do not cause disease, are delivered to motor nerves through intravenous drips.