Rs 17 crore for a single dose: Why Zolgensma is world's most expensive medicine

Zolgensma injection, a one-time gene therapy used for children under two suffering from spinal muscular atrophy (SMA), is once again in the headlines. Though it is not approved in India, it can be imported through a doctor's recommendation and government approval. Apart from benefit of the drug, one of the world's most expensive drugs which costs Rs 17 crore for a single jab, its price is often is in the spotlight. Karnataka Chief Minister Siddaramaiah recently reached out to Prime Minister Narendra Modi, seeking help for the medical treatment of a 15-month-old boy battling spinal muscular atrophy.

Zolgensma, developed by Swiss pharmaceutical company Novartis, is aimed for treatment of SMA, a rare genetic disease. According to Cleveland Clinic website, spinal muscular atrophy (SMA) affects nerves and muscles, causing muscles to become increasingly weak. It mostly affects infants and children but can also develop in adults. Around 10,000 to 25,000 children and adults, according to estimates, are living with SMA in the United States.

"Zolgensma delivers a functional copy of the SMN gene into motor neuron cells, improving muscle movement and function in children with SMA," Dr N Varsha Monica Reddy, consultant paediatric neurologist, Yashoda Hospitals, Hyderabad, told Press Trust of India..


"The gene encodes the SMN protein - found throughout the body, which is critical for the maintenance and function of motor neurons. In the absence of enough functional SMN protein, motor neurons die, leading to debilitating and often fatal muscle weakness," she added.

Medical experts say that with relatively very few patients buying these drugs, the cost remains high as the costs, including high R&D costs, gets passed to a few.

Vas Narasimhan, Chief Executive Officer of Novartis, in LinkedIn post said that gene therapies represent a medical breakthrough that offers hope for deadly genetic diseases with a single dose. "But these breakthroughs present a big challenge—how do you value and pay for a one-time therapy that provides a lifetime of benefits to a patient?" he said.

Paediatrician Vibhu Kawatra, a visiting consultant at Delhi's Rainbow Children's Hospital, told Press Trust of India, that Zolgensma's cost at approximately Rs 17 crore in India, without (minus taxes) is due to the extensive research and development involved.

"The limited market size and its potential to save lives contribute to the high price. Despite its initial expense, experts argue that Zolgensma's long-term benefits may offset healthcare costs associated with SMA treatment and care," Kawatra, , told PTI.

According to Novartis website, the drug is approved in 45 countries, with more than 2 500 patients treated globally across clinical trials, managed access programs, and in the commercial setting. The company itself has provided nearly 300 children in 36 countries with our gene therapy free of charge, according to its website.