One time treatment over 14 million yuan! Novartis will provide the world's most expensive medicine free of charge by drawing lots

More than half a year ago, zolgensma, Novartis's gene therapy, sparked a heated debate over its high price Now, Novartis has announced that it will provide the world's most expensive drug free of charge by drawing lots Recently, Novartis said that from January 2 next year, avexis, a company that develops zolgensma, will distribute 50 doses of zolgensma single dose treatment to infants under 2 years old by June The free drug plan is for countries that have not yet approved zolgensma, and avexis plans to distribute up to 100 doses per year according to production capacity It is understood that the drug will be distributed in the form of lottery According to the plan, doctors will submit treatment requests to the company on behalf of patients, and an independent committee will conduct a two-week lottery for eligible infants If the patient is not selected, they will enter a subsequent draw until their eligibility expires Once zolgensma is approved by a country or region, Novartis will terminate the draw locally In May this year, the FDA approved zolgensma for the treatment of spinal muscular atrophy (SMA) patients under 2 years old who carry mutations on two SMN1 alleles encoding motor neuron survival protein (SMN) It carries healthy genetic materials into human somatic cells by using engineering virus to replace the defective or mutated genes that cause the disease, and is administered intravenously (s Pinraza is intrathecal), which is the first and only gene therapy approved by FDA for SMA Zolgensma is a one-time treatment, priced at US $2.215 million, equivalent to more than 14.6 million yuan, and is known as the most expensive drug in the world At that time, Novartis said that it was ready to reduce the initial burden of patients by means of five-year installment Within five years, if the treatment was invalid or the patient died, Novartis would refund and provide advance discount to payers willing to sign standardized insurance terms Previously, Biogen's spinraza is the only drug approved for the treatment of spinal muscular atrophy Unlike Novartis, spinraza is a life-long treatment, with a price of 750000 dollars in the first year, 375000 dollars in each year, 2.25 million dollars in five years and 4.5 million dollars in ten years The high price has always been a unique label of gene therapy Also this year, Bluebird announced the price of its gene therapy, zteglo (lenti globin, approved in the European Union), which is the second most expensive drug in the world after zolgensma Zynteglo is used to treat non β 0 / β 0 transfusion dependent β - thalassemia (TDT) patients aged 12 years and over They need regular blood transfusion to treat the disease and there is no matching stem cell transplant donor Lentiglobin is the first product approved by Bluebird biology and the first approved TDT gene therapy in the world.