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Zhiyaoke reported
More than half a year ago, Zolgensma, the gene therapy of Novartis , caused heated discussion because its pricing was so high. Now, Novartis announced that it will provide this world's most expensive drug for free through lottery.
Recently, Novartis said that starting from January 2 next year, AveXis, a company under Novartis, will distribute 50 doses of Zolgensma single doses to babies under the age of 2 by June. This free drug program is for countries that have not yet approved Zolgensma, and AveXis intends to distribute up to 100 doses per year based on production capacity.
It is understood that the drug will be issued in the form of a lottery, and according to the plan, the doctor will submit a treatment request to the company on behalf of the patient, and an independent committee will draw lots for eligible babies every two weeks.
If patients are not selected, they will enter the subsequent draw until the qualification expires. Once a country or region approves Zolgensma, Novartis will terminate the lottery plan locally.
In May this year, the FDA approved Zolgensma for the treatment of spinal muscular atrophy (SMA) patients with mutations on two SMN1 allele encoding motor neurons. They carry healthy genetic material into human cells by using engineered viruses to replace defective or mutational genes that cause the disease and administer intravenously (Spinraza is intrathecal), which is also the first and only gene therapy approved by the FDA for the treatment of SMA.
Zolgensma is a one-time treatment, priced at US$2.125 million, equivalent to more than RMB 14.6 million, and is known as the most expensive drug in the world.
At that time, Novartis said that they were planning to reduce the initial burden on patients by using five-year installments. If the treatment is ineffective or the patient dies within five years, Novartis will refund and provide advance discounts to payers who are willing to sign standardized coverage terms. What diseases does
"sky-high drug" treat?
SMA is one of the most common lethal neuromuscular diseases and the number one genetic cause of infant death, caused by defects in the single gene motor neuron survival gene SMN1. According to the time and severity of the disease, SMA can be divided into types 1, 2, 3 and 4. About 60% of SMA patients are type 1 and the most severe type.
It is estimated that the incidence of SMA is 1/6000~1/10000, which means that one in every 10,000 newborns is affected, and 90% of the sick babies live to be 2 years old.
Figure | Spinal muscular atrophy (Source: evolving-science.com)
Previously, American pharmaceutical company Biogen's Spinraza is currently the only drug approved for the treatment of spinal muscular atrophy. Unlike Novartis, Spinraza is a lifelong treatment, with a price of US$750,000 in the first year, and a price of US$375,000 in the next year, a cost of US$2.25 million in the five years, and a cost of US$4.5 million in the ten years.
What is gene therapy?
gene therapy (gene therapy) refers to the introduction of exogenous normal genes into target cells to correct or compensate for diseases caused by defects and abnormal genes to achieve therapeutic purposes.
also includes technical applications in genetically modified areas, that is, inserting exogenous genes into appropriate receptor cells of the patient through gene transfer technology, so that the products made by exogenous genes can treat a certain disease.
Broadly speaking, gene therapy may also include measures and new technologies for treating certain diseases taken from the DNA level.
On October 19, 2017, the US government approved the second therapy based on the modification of patients' autoimmune cells (yescarta gene therapy) to treat specific lymphoma patients.
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