Recently a drug named Zolgensma, has been approved by Britain. The drug is recently approved by National Health Service (NHS) of UK. Zolgensma is the worlds most expensive drug till now. This drug is being said to cure the spinal muscular atrophy SMA in babies.
The cost of each ‘World’s Most Expensive Drug’ has been said to be about spinal muscular atrophy £1.79 million that means ₹18 crore. The price is highest of all the drugs found till now. This drug has been manufactured by the Novartis Gene Therapies.
As per the expectations the demand will be higher for the drug, because it has found significant effects on the genetic disorder called spinal muscular atrophy.
The chief executive of the National Health service England said in an official statement that “This deal is a life-changer for youngsters with this cruel disease and for their families. Spinal Muscular Atrophy is the leading genetic cause of death among babies and young children, which is why NHS England has moved mountains to make this treatment available, while successfully negotiating hard behind the scenes to ensure a price that is fair to taxpayers.”
Spinal muscular atrophy is a severe genetic disorder. Children who suffer from it, most of them have less life expectancy.
What is spinal muscular atrophy (SMA)?
Spinal muscular atrophy (SMA) is a rare disorder which is a neuromuscular type of disorder. Spinal Muscular atrophy often called SMA results in the progressive muscle wasting and loss of motor neurons. SMA usually affects the infants or children of early age. The treatment should be done in the early stage because if it left untreated then it can lead to the infant’s death with genetic reasons.
SMA may appear again in the life later and can have a milder course of the disease. As this is neuromuscular disorder it affects the muscles. Some common features found are progressive weakness of the muscles in the body, including legs, arms, respiratory muscles too. Spinal Muscular Atrophy can lead to the associated disorders like difficulty in the swallowing, poor head control, joint contractures, and scoliosis etc.
Several symptoms for the SMA are Difficulty in swallowing food
Weakness of the muscle thus inability to walk or stand
Numbness over certain areas
Hypotonia -diminished muscle tone
Involuntary contractions or twitching of muscles
Decreased or absence deep tendon reflexes
The children diagnosed with the type 1 Spinal Muscular Atrophy (SMA) have life expectancy of 2 years only. That’s why it is a severe disorder and rare too.
Zolgensma is the ‘World’s Most Expensive Drug’ till now. This drug is being said to cure the spinal muscular atrophy SMA. The cost of the drug is £1.79 million that means ₹18 crore per dose.
About the ‘World’s Most Expensive Drug’:
Recently a drug named Zolgensma, has been approved by Britain which treats the spinal muscular atrophy. Zolgensma treats Spinal Muscular Atrophy (SMA) which is a fatal genetic disease.
The data of this drug has suggested that the administration of this drug has been found to improve the function of the motor nerves in the children diagnosed with type one spinal muscular atrophy. This drug has shown effects such as the babies began to breath without a ventilator, crawl and walk, sit up of their own etc. etc. And the point to be noted that this happened in just a single infusion.
As said by NHS, the treatment is given as single dose as infusion intravenously. The mechanism of action of the drug is of targeting missing genes in the patient. The dose contains replica of the missing gene SMN1. The active ingredient called onasemnogene abeparvovec, enters into the nerves. It restores the missing gene. The restoration causes production of the essential protein for the nerve function and muscular movement. And that’s hoe the babies recover from the spinal muscular atrophy, NHS said.
Cost of the drug
The drug called Zolgensma, has been approved by Britain recently with the highest cost in the world per dose. The cost of the single dose is £1.79 million that means ₹18 crore in Indian rupees. Which means the 2464200.00 United states dollars in the current situation.
NHS England has said that they are taking and have taken to make this treatment available easily. The cost of 18 crore rupees makes the zolgensma the most expensive drug in the world.
The treatment over the spinal muscular atrophy has been found with this ‘World’s Most Expensive Drug’ named zolgensma, with the cost of the 18 crore Indian rupees approved by Britain recently.