Do you know how expensive the most expensive drug in the world is? A needle of 2.125 million US dollars (equivalent to RMB 13.48 million), do you think it is outrageous enough? And this “most expensive drug in history” will now land in China.
Recently, the Center for Drug Evaluation (CDE) of the State Food and Drug Administration of China announced thatThe clinical trial application of Novartis’ AAV gene therapy drug Zolgensma (OAV101 injection) for the treatment of Spinal Muscular Atrophy (SMA) in China has been granted an implied license for clinical trials.
Previously, on October 21, 2021, the clinical trial application submitted by the drug was accepted.
At present, Zolgensma has been approved in nearly 40 countries and regions around the world. Among them, there are many countries that include it in medical insurance.Zolgensma was included in Japan’s medical insurance in 2020, and patients only need to pay 30% of the cost; it was later included in the UK’s national medical service system in March 2021.
Many netizens have also learned about the disease “gradual freezing”, and its “brother disease” is SMA, which is as cruel as “gradual freezing”. SMA patients will continue to lose the corresponding motor function, but “gradually freezing” “Frozen people” is generally an adult onset, while SMA is generally onset in infants and young children, and the earlier the onset of SMA, the more serious it is.
According to the data, spinal muscular atrophy (SMA) is a kind of disease caused by the degeneration of motor neurons in the anterior horn of the spinal cord, resulting in muscle weakness and muscle atrophy. The disease can be caused by a variety of genetic mutations, but generally refers to an autosomal recessive disorder caused by mutations in the survival motor neuron gene 1 (SMN1).
On May 24, 2019, the US FDA approved Novartis’ first gene therapy, Zolgensma, for the treatment of SMA patients under 2 years of age.Patients only need to receive a single intravenous injection to express SMN protein in cells for a long time, achieving long-term remission or even cure.
Zolgensma is a special medicine for the “SMA” disease, but the sky-high price of $2.125 million for one injection has also made it controversial, and it is considered to be a threat to the child’s life.
The drug has also been controversial, with critics saying it’s a runaway pricing exercise, while supporters say Novartis has found a cure for the rare disease that could save desperate families.
Regarding the doubts of society and public opinion, the CEO of Novartis once said: Those critics did not really think about how our medical system works.
We do transplants on patients, which cost $3 million to $5 million per patient, but are far less effective than these drugs.The CEO said that if the therapy is performed only once, the cost will be cheaper than the current therapy.
However, three SMA therapies have been approved globally, in addition to Zolgensma, Biogen’s Spinraza and Roche’s Evrysdi, the latter two have been approved in China.
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