The most expensive drug in the world is Zolgensma, the price of the drug is £1.79 million (Rs.18 crore) per dose. NHS England authorised Zolgensma for the treatment of babies with a rare deadly genetic disease.

Zolgensma, a life-saving medicine, can enable mobilitiy in newborns and young toddlers suffering with a rare genetic disease. Zolgensma is an adeno-associated virus vector-based gene therapy for the treatment of spinal muscular atrophy in children under the age of two.

SMA is a rare and sometimes deadly genetic illness that causes paralysis, muscle weakness, and progressive loss of movement. The one-off gene therapy treats SMA. Babies born with severe type 1 SMA, the most common form of the disease, have a two-year life expectancy.

After a single infusion therapy, Zolgensma, manufactured by Novartis Gene Therapies, has helped babies achieve milestones like breathing without a ventilator, sitting up on their own, crawling, and walking.

According to the latest research, Zolgensma can help young children with type 1 SMA and prolong their life span.

Recently, Ayaansh Gupta, a three-year-old from Hyderabad with a rare spinal muscular atrophy (SMA) was given Zolgensma, cost Rs 16 crore for his gene therapy treatment. The drug was imported from the US with donations and administered to Ayaansh at Rainbow Children’s Hospital in Secunderabad on June 9.

SMA affects one out of every 10,000 children worldwide, with over 800 children in India are now suffering from the disease, and thrice as many children are dying before reaching their second birthday.