Sorgensma, an expensive treatment for intractable diseases of infants, is covered by medical insurance at 14:10 on February 26
"Solgensuma", a treatment for intractable diseases of the whole body such as young children, whose muscle strength is weakened, is approved by the government and is expected to be covered by medical insurance. While high effects are expected, the United States is said to be the world's highest drug with a price exceeding 200 million yen, and the Ministry of Health, Labor and Welfare will discuss what kind of price it will set in Japan in the future.
The drug is expected to receive approval for the treatment of intractable disease, spinal muscular atrophy, for which Onasemnogen Abeparbobek and product name Zolgensuma have been filed by the pharmaceutical company Novartis Pharma.
The policy was approved by the MHLW's Council on 26th.
Spinal muscular atrophy is an intractable disease in which a part of the gene is mutated and the whole body's muscular strength is reduced. If it develops in a newborn infant, 90% of the children will die by the age of 2 It is said that a respirator is needed.
"Solgensma" improves the function of nerves and muscles by introducing a virus with a specific gene into the body.
According to the Ministry of Health, Labor and Welfare, a single infusion is effective, and overseas clinical trials show that all infants administered up to 9 months of age who grew up to 2 years of age without the need for ventilators were administered.
Approximately 20 newborns develop the disease each year in the country, and Sorgensma is expected to cover infants under 2 years of age.
In the United States, it was approved last May, but it costs over 200 million yen at a time, and is said to be the world's highest treatment.
"Solgensma" is expected to be officially approved after a month or so, and the Ministry of Health, Labor and Welfare will discuss what price it will set in Japan.