The sky -high drug of 14 million yuan has caused two children to die

On August 11, Novartis announced that it was used to treat two children's death by gene therapy drug Zolgensma, which was used to treat spinal muscle atrophy (SMA). The cause of death was acute liver failure. This is the first time that this "magic medicine" has happened.

ZOLGENSMA is known as "the most expensive medicine in the world", with per capita treatment costs of nearly US $ 2.1 million, equivalent to RMB 14 million. After listing in the United States in May 2019, because it focuses on the treatment of SMA "one shot", Novartis has always believed that high pricing is rational.

At present, Zolgensma has conducted clinical trials in China and has not been listed. The above two deaths occurred in Russia and Kazakhsta, respectively, and the two countries did not allow Zolgensma to be listed.

Nuohua said to the Jianzhi Bureau: "Both cases are used in the way that the local health department is allowed. Among them, patients in Central Asia are treated through a early and sexual plan of Kazakhstan."

In response to the progress of Zolgensma in China, Novartis said to the Jianzhi Bureau: Related clinical trials are being promoted as planned.

A bad response of the warning, in China, is clinical in China

According to Novartis, the two children died due to acute liver failure. The death time was about 1-10 days after the use of Zolgensma about 5-6 weeks after starting to reduce corticosteroids.

In this regard, Nuohua announced that liver failure is a clinical safety event, but it is not a new security signal.

Zolgensma is not originally created by Novartis, but a product obtained by Avexis in April 2018 for $ 8.7 billion. But since its listing, this medicine has been deepening.

It is understood that Zolgensma is mainly used to treat patients with rare disease SMA under 2 years of age. However, on the pharmaceutical manual published by the FDA official website, Zolgensma was listed in a black frame warning: the drug may cause acute severe liver injury, acute liver failure, and elevated ammononase.

Prior to the emergence of Zolgensma, the most expensive drug for the treatment of SMA was the "sky -high medicine" Nosina sodium with 700,000 needles. However, Nosina sodium needs to be injected every year, and Zolgensma is "one -needle for life". Although the price is as high as $ 2.1 million, if it can really be able to pay for it once and for all, many patients are willing to pay.

Since landing in the US market in 2019, Zolgensma has been approved to be listed in more than 40 countries and regions including the European Union and Japan, with a total of more than 2,300 patients. In 2021, Zolgensma's global sales were about $ 1.4 billion, an increase of 47%year -on -year.

Prior to this, there have never been cases that have caused liver failure to cause liver failure due to Zolgensma.

After the death, Novartis immediately communicated with the health authorities to update the product label of ZOLGENSMA, which clearly stated that the drug had had a fatal acute liver failure. But at the same time, it still believes that the overall safety/efficacy of Zolgensma is still in good condition.

However, Novartis has been prepared for the worst.

Novartis said that the health authorities in all countries and regions sold in Zolgensma have been notified. It may affect the promotion and application of drugs in the world in the future.

In China, ZOLGENSMA opened phase III clinical trials in April this year; in early July, the drug intended to be used for the treatment of SMA were included in the breakthrough therapeutic drugs. Novartis said that related clinical trials are being promoted as planned.

The dilemma of rare diseases can only take risks for the effect?

During the medical insurance negotiations in 2021, the "700,000 pins" of the sky -high medicine was cut to 33,000 yuan. Many parents of parents have been looking forward to Zolgensma's entry into China since then.

Solid muscle atrophy is a "number one killer" with high residual, highly dead infant, and entered the first rare disease catalog in 2018. At present, only three drugs around the world can be used to treat the disease: Bo Jian's Nosna sodium, Roche's Lispra and Novarton's Zolgensma.

Relatively speaking, Zolgensma is the most expensive. Because the drug starts with gene therapy, the AAV carrier is used to inject the patient's lack of SMN1 gene into its body and cause a normal SMN1 protein. So in theory, this medicine only needs to be used once. The other two drugs need long -term continuous medication.

Zolgensma officially obtained clinical application acceptance of the China Pharmaceutical Supervision Bureau on October 21, 2021. This made the two peer companies jealous.

In December 2021, Nosnina Sodium participated in medical insurance negotiations, from 700,000 to 33,000 yuan. , Actively lay out to enter medical insurance.

The death incident may affect the approval process of Zolgensma.

Objectively speaking, the adenovirus carrier (AAV) gene therapy used by Zolgensma has controversy. Shortly after this drug was launched, the original researcher Avexis had disclosed to the FDA that the gene therapy had some data control problems.

The AAV gene therapy of the peers is also unhappy.In September 2021, Anstelai announced the suspension of its AAV gene drug AT132 for the clinical trial of X chain muscle disease (XLMTM), because the drug caused the death of four patients, and all four patients had liverquestion.Also last year, American gene therapy company Adverum Biotechnology Inc. suspended its AAV gene therapy drug ADVM-022 for the treatment of diabetic retinopathy macular edema, because the patient had a serious adverse reaction to the therapy.

Precision treatment is the development direction of modern medicine, especially for rare diseases, and is also a preference for many companies.However, all fresh areas need to be trial and error and exploration. When the inevitable situation of drug development such as failure and death appears, how the market should think of exploration and failure, which is related to the future of rare new drug development.

Writing ｜ Nicotinamide

Edit ｜ Jiang Yun Jia Ting

Operation | Twenty -thirty

Illustration | Visual China

#AAV gene therapy ## The most expensive medicine in the world#

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