The most expensive medicine in the world

England’s Ministry of National Health approved the most expensive drug in the world. The name of the drug is Zolgensma. The price of one dose of which will surprise anyone. One dose of this medicine costs 18 crore rupees.

It is a medicine for a rare disease called Spinal Muscular Atrophy or SMA. This disease is seen in the body of one in a million. Whichever happens, along with his life, his family is in dire danger. Experts say this rare genetic disease cripples people as well as weakens the body’s muscles. People with SMA even lose the ability to walk. Many times, it also causes the death of people.

Zolgensma brings Novartis’ gene therapies to the market to treat the rarest form of SMA. The cost of one dose of the drug is said to be 18 crore rupees. England’s Ministry of National Health has given permission to sell it in the market. Experts say that mainly children are born with the first type of SMA. It has been reported that it may cause their loss of life later. However, it is also believed that those children can return to normal life with the application of Zolgensma.

History of Medicine

Onasemnogene abeparvovec, developed by US biotechnology startup AveXis, which was acquired by Novartis in 2018, is based on research conducted at the Institut de Myologie in France. The US Food and Drug Administration has granted onsemnogene abeparvovec-GOI several designations, including fast track, breakthrough therapy, priority review, and orphan drug designation.

In May 2019, onasemnozine abeparvovec received US FDA approval as a treatment for children under two years of age. As of 2019, treatment has been reimbursed in Qatar and Israel. In March 2020, it received regulatory approval in Japan with the same labelling as in the US.

In August 2020, onasemnogene abeparvovec received Brazilian regulatory approval from the Brazilian Health Regulatory Agency. Subsequently, it was approved for medical use in Canada in December 2020, in Australia in February 2021, and in Russia in December 2021. Onasemnogen abeparvovec was approved in April 2023, according to the Health Sciences Authority of Singapore register.

This medicine is used to treat any disease

Onasemnogen abeparvovec, sold under the brand name Zolgensma, is a gene therapy used to treat spinal muscular atrophy, or SMA. A rare disease that reduces muscle function in children. It works by providing a new copy of the SMN gene that makes the SMN protein.

Onasemnogene abeparvovec is developed to treat spinal muscular atrophy, a disease associated with mutations in the SMN1 gene on chromosome 5q and diagnosed mainly in young children that causes progressive loss of muscle function and often leads to death. The drug is administered as an intravenous infusion.

Why is the Zolgensma drug so expensive?

The reason Zolgensma is so expensive is the price that Novartis has decided is worth it because it “dramatically changes the lives of families affected by this devastating disease” and the claimed cost of bringing the new drug to market.

Novartis’ CEO, Vas Narasimhan, argues that gene therapies represent a medical breakthrough in that they offer the hope of curing fatal genetic diseases with a single dose. In some cases, the alternative is multi-dose treatment with incremental improvement. That is why this medicine is so valuable.

How does Zolgensma work?

Zolgensma treats the genetic root cause of SMA by replacing the missing or dysfunctional SMN1 gene with a new, functional copy of the human SMN gene. It uses a vector that can get a new, functional SMN1 gene into the body. In this case the vector is a virus called AAV9 that has had its DNA removed and replaced with the SMN1 gene. This type of virus does not make you sick but can quickly travel through the body to motor neuron cells and deliver new genes.

Zolgensma sits inside the nucleus of motor neuron cells and tells motor neuron cells to make new SMN1 protein. Once the genes reach their destination, the vectors break down and are excreted from the body and do not become part of the baby’s DNA.

Zolgensma drug side effects

A very serious allergic reaction to this drug is rare. However, if you notice any signs of a serious allergic reaction, including: rash, itching/swelling (especially of the face/tongue/throat), severe dizziness, trouble breathing, get medical help right away. This is not a complete list of possible side effects.

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