On Tuesday, The UK’s National Health Service (NHS) approved the world’s most expensive drug that can prevent a rare genetic disorder. The drug, called Zolgensma and manufactured by Novartis Gene Therapies, costs Rs 18 crore (£1.79 million) per dose, according to the official statement NHS England. Yes, you read it right!Also Read - Luxury Penthouse in Hong Kong Sells For Whopping Rs 420 Crore, Most Expensive Flat Ever Sold In Asia!
As per the official statement of NHS England’s chief executive Sir Simon Stevens, “This deal is a life-changer for youngsters with this cruel disease and for their families.” He further added, “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.”
What is Spinal Muscular Atrophy?
Spinal muscular atrophy (SMA) is a rare disease that most often affects babies and children and makes it hard for them to use their muscles. The fatal genetic disease causes paralysis, muscle weakness and progressive loss of movement, caused by a loss of specialized nerve cells, called motor neurons that control muscle movement. It strikes about 400 babies born in the U.S. each year. Babies born with severe type 1 SMA – the most common form of the condition – have a life expectancy of just two years.
How will Zolgensma treat SMA?
Zolgensma will be used for infants and kids who are suffering from SMA. The treatment involves a single intravenous infusion that replaces the missing gene SMN1. It leads to restore the gene as the ingredient onasemnogene abeparvovec enters the nerves. The gene then produces essential proteins for nerve function and controlls muscle movement.
Zolgensma can help babies breathe without a ventilator, sit up on their own, and crawl and walk after a single infusion treatment, as per previous studies. The latest data suggested that Zolgensma can provide rapid and sustained improvement in motor function for young children with type 1 SMA and prolong their lives.