Zolgensma - World's Most Costliest Drug to Treat Rare Genetic Disorder

Did you ever thought about the World's Costliest Drug ? If not then let me tell you it is Zolgensma which is used to treat a rare genetic disorder called Spinal Muscular Atrophy(SMA). The Cost of Drug is estimated to be around Rs16 to Rs18 crore in Indian Rupees. Since this Drug is not manufactured in India, it has to be imported from USA. There are only handful of nations which makes this Drug. More on Zolgensma official website.

What is Spinal Muscular Atrophy

It is a rare genetic condition arises mostly in children due to one missing gene which is responsible for creating a protein that controls body essential functions like breathing, walking, speaking and swallowing. This missing gene which is also known as Survival Motor Neuron 1(SMN1) gene is given through the drug Zolgensma.

Zolgensma - World's Most Costliest Drug to Treat Rare Genetic Disorder

Zolgensma - World's Most Costliest Drug to treat Rare Genetic Disorder

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Zolgensma is a gene therapy approved by US Food and Drug Administration(FDA) for children less than 2 years old suffering from Spinal Muscular Atrophy Disorder. It is only required to be given as one time dose intravenously. Infusion takes around 60 minutes to complete.

Who manufacture this Drug

Zolgensma is manufactured by Swiss multinational pharmaceutical company called Novartis Gene Therapies. It was first approved on May 24, 2019 by the US Food and Drug Administration and by the United Kingdom's National Health Services (NHS) on March 9, 2020.

Why It is So Costly

The price of the drug is high due to low incidence of this disease. According to a report, the prevalence of this genetic disorder is about 1 in 10,000 children. This rare genetic disorder attracts significant amount of research and study which results in this much high cost. Apart from this, the cost of the drug also get negatively impacted when it has to be imported from countries like US where medicine cost is almost four times the cost it has in India. All this factors makes this drug as one of the costliest drug.

How Does it Work 

Zolgensma (onasemnogene abeparvovec-xioi) is an adeno-associated virus 9(AAD 9) vector-based gene therapy that target the genetic root cause cause of SMA by replacing the function of the missing or nonworking SMN1 gene with a new, working copy of a human SMN gene.

It is designed to produce SMN protein, which preserves motor neuron cells and, in turn, stops the progression of SMA and sustains the remaining muscle function needed for children to survive. It is a one time use genetic therapy as the second time antibody will resist the effect of this drug.

Efficacy of this Drug

The safety and efficacy of ZOLGENSMA (onasemnogene abeparvovec-xioi) have been established in 3 clinical studies (2 completed and 1 ongoing) and 1 long-term follow-up study. ZOLGENSMA has been shown to stop the progression of spinal muscular atrophy (SMA). In one study, about 91% (20/22) of patients were alive and did not need permanent breathing support at 18 months of age. In the natural history of SMA, about 25% of patients with SMA Type 1 were alive without permanent breathing support at 14 months of age.

Safety Information

Sometimes Zolgensma can cause acute serious liver injury. Liver enzymes could become elevated and may reflect acute serious liver injury in children who receive Zolgensma. Hence it is always recommended to keep the patient under Doctor observation after injecting this drug.

Any Other SMA Drugs

Apart from Zolgensma , there are two other alternatives available in the market - Biogen’s Spinraza and recently approved, Roche and Genentech’s Evrysdi. Spinraza was approved by US FDA in December 2016. It is an injection administered into the fluid around Spinal Cord. Similarly, Evrysdi was approved by US FDA in August, 2020. It has to be taken orally once daily after a meal at approximately the same time each day.

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