Recently, the official website of the Drug Evaluation Center of the State Drug Administration shows that the clinical trial application submitted by Zolgensma, an AAV gene therapy drug developed by Novartis Pharmaceuticals for the treatment of spinal muscular atrophy (SMA), has been implicitly approved in China.
Source: Network
Zolgensma (OAV101 injection) is an AAV gene therapy drug for spinal muscular atrophy, priced at a price of US$2.125 million (equivalent to about 13.48 million yuan) per shot, and has been called "the most expensive drug in history". At present, the drug has been approved in nearly 40 countries and regions around the world. However, the only countries that include the drug in medical insurance are Japan and the United Kingdom.
What is spinal muscular atrophy?
Spinal muscular atrophy (SMA) is a motor neuron disease in which motor nerve cells fail to function properly, causing patients to gradually lose motor function, and in severe cases, leading to inability to breathe and swallow, and the disease has pathogenic genes that can be inherited. According to statistics, the incidence of SMA in newborns is between 1 in 6,000 and 1 in 10,000.
One injection for long-term remission or cure
In 2019, Zolgensma, a gene therapy developed by Novartis Pharmaceuticals, was approved for marketing by the US FDA, which means that Zolgensma is the first gene therapy approved for the treatment of SMA patients under 2 years of age.
It is reported that when SMA patients are treated through this gene therapy, they only need to be injected intravenously once to achieve long-term remission or even cure.
Sky-high prices for drugs are controversial
Although Zolgensma's therapeutic effect is remarkable, a single injection can achieve long-term remission or cure, but the consequent high price is unbearable for the average person and is considered a pricing runaway behavior. Novartis Pharmaceutical has also been questioned as a result, and has been criticized for not respecting life and threatening life.
In the face of public skepticism, Novartis responded: "Critics are not really thinking about how our health care system works. Each SMA patient can cost $3 million to $5 million for a transplant, but it's far less effective than these drugs. This treatment can be relieved or cured for a long time only once, and the cost of gene therapy will be cheaper than the current cost of transplant surgery. ”
Every small group should not be abandoned
The emergence of specific drugs is a hope for the families of children, and now there are drugs available, but the price that comes with it has become another major problem in front of rare diseases.
At present, the only three types of treatment approved for the treatment of SMA in the world are Novartis Zolgensma, Bojian Spinraza and Roche Evrysdi, and the latter two have been approved in China.
The pricing of Spinraza in China is also close to 700,000 bottles, which need to be injected repeatedly every year, and are completely self-funded drugs.
Previously, the phrase "every small group should not be abandoned" in the 2021 medical insurance negotiations believes that everyone remembers, and it is precisely because of this negotiation that Spinraza has dropped from a single needle price of 700,000 yuan to more than 30,000 yuan, which has made many rare disease patients see hope.
In response to the decline in Spinraza medical insurance negotiations, some insiders analyzed that "the number of SMA children in China is large, the drug market is very large, and Bojian chose medical insurance cooperation to vote in China in the strategic choice and seize the Chinese market."
In recent years, more and more rare disease drugs have been included in the national medical insurance list. According to statistics, after the medical insurance negotiations in 2021, 58 drugs involving 28 rare diseases have been included in the medical insurance list, and it is hoped that in the near future, 13 million doses of Zolgensma gene therapy will also be successfully negotiated by the state into medical insurance, benefiting more children with SMA in ordinary families.