2021/02/21 19:44 60.22 million yuan per stitch!Injecting the most expensive drug on the ground



The Swiss pharmaceutical company Novartis' new drug Zolgensma can be used to treat infants and children under two years of age suffering from the rare spinal muscular atrophy (SMA), but it costs US$2.125 million for a dose, making it the most expensive drug in history.  (Associated Press)

The Swiss pharmaceutical company Novartis’ new drug Zolgensma can be used to treat infants and children under two years of age suffering from the rare spinal muscular atrophy (SMA), but it costs US$2.125 million for a dose, making it the most expensive drug in history. (Associated Press)

2021/02/21 19:44

[Compiling Chen Chengliang/Comprehensive Report]The Russian Satellite News Agency (Sputnik) reported on the 20th that a 7-month-old boy in Yekaterinburg, Russia’s fourth largest city, was diagnosed with spinal muscular atrophy. , Referred to as SMA), after which the doctor injected a dose of Zolgensma, the world’s most expensive drug with an asking price of US$2.125 million (approximately NT$60.22 million). ) The charitable foundation under his name raised it.

According to the report, similar drugs in Russia have no long-term effect on SMA. Patients need to use the drug for life, and Zolgensma can be used for life after a single injection of Zolgensma. Zolgensma is a gene replacement therapy that can replace diseased genes to restore health.

The probability of infants and young children suffering from hereditary SMA is about one in ten thousand, and 50 to 70% of them will develop the most serious type I, often with symptoms such as gradual paralysis, difficulty breathing and swallowing within a few months, and 90% You will lose to the disease at the age of two, and even if you survive, you may have to rely on respirators for your living.

Zolgensma, developed by Novartis, a Swiss pharmaceutical company, is used to treat SMA patients under 2 years of age, including those who have not yet developed symptoms. The drug uses the latest gene therapy to directly implant healthy DNA (deoxyribonucleic acid) into human cells to repair defective genes and achieve the purpose of treatment. The most expensive drug in history was approved by the U.S. Food and Drug Administration (FDA) in May 2019.

The FDA pointed out that the earlier Zolgensma treatment is started, the better the effect, but it cannot recover the physical damage that has occurred before treatment. The most common side effects are increased liver enzymes and vomiting, which must be observed for several months after treatment.

Novartis claims that even if the price of the drug hit a record high in history, the one-time Zolgensma treatment is relatively cheaper than the current SMA drug Spinraza, which costs about US$4 million (about NT$113.55 million) for 10 years. In addition, when it is listed in the United States, Novartis not only allows the insurer to amortize the payment for five years, but can also sign an agreement to pay only when the drug is effective. The Taiwan Food and Drug Administration has not yet approved this new drug.



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