India approves Zolgensma, one of world’s costliest drugs. Why it’s sparked hope, but also concern

Zolgensma is the only drug that promises a cure for Spinal Muscular Atrophy, provided it is administered to affected children before the symptoms appear.

Zolgensma, a global blockbuster, can cure Spinal Muscular Atrophy under certain conditions | Photo Credit: Novartis Pharmaceuticals

New Delhi: For the last three months, a mother in Ratnagiri, Maharashtra, has been desperately trying to raise funds for accessing Zolgensma—not only a global blockbuster but also a super-expensive gene therapy for the rare genetic disorder, Spinal Muscular Atrophy (SMA)—for her daughter, born with the severest form of the disease.

Her daughter, who is five months old, has limited motor movement and has now started having trouble feeding—a sign that her muscles are deteriorating fast. The therapy, one of the costliest in the world with a listed price of Rs 17.7 crore in the United States, is the only medicine that offers hope of the baby’s survival in just one shot.

However, time is ticking by, and so far, the mother, a homemaker whose husband is a small-time trader, has managed to raise only Rs 91 lakh for the medicine by the Swiss pharma giant, Novartis. 

“I heard that the drug, if offered quickly, can arrest the progression of the disease…I contacted Novartis, and the company has agreed to give us the drug under an EMI plan, provided we manage to pay Rs nine crore in the first instalment, but raising this huge amount is not easy, despite the best of our efforts,” the mother told ThePrint over a phone call from Ratnagiri.

Two years ago, she had lost her 2.5-year-old son to the same disorder. 

Last week, government sources told ThePrint that the Central Drugs Standard Control Organisation (CDSCO) granted regulatory approval to Zolgengsma, paving the way for its commercial launch in India. 

Though Novartis is yet to decide the price of the therapy for Indians, experts say that it will come down only marginally and that it may still be the costliest drug in the Indian market. The development, however, has come as a ray of hope for desperate parents as they expect that its India launch will bring down the cost of the therapy.

Zolgensma is the only drug that promises a cure for the condition, provided it is administered to affected children before the symptoms appear.

For Spinal Muscular Atrophy type 1, the severest and most common form of the disease, the therapy is approved for babies till the age of two years and can arrest the progression of the disorder, if administered within the cut-off age. 

Annually, in India, an estimated 4,000 babies are born with Spinal Muscular Atrophy, a severe and progressive genetic neuromuscular disease, robbing individuals of essential physical abilities, such as walking, eating, and breathing—the leading genetic cause of death among children worldwide.

Since 2020, when the drug was first launched in the US, nearly 60 Indian babies have received imported Zolgensma, either through a Novartis-run humanitarian programme under which it offered the drug to affected babies globally through a lottery system, or even crowdfunding. 

Those advocating for the rights of rare-disease patients say the India launch will stop the time-consuming and acutely cumbersome process of importing the drug, and hopefully, bring down its cost.

“In case of severe SMA, time is of immense value as the babies get sicker and sicker every day, and sometimes, it takes months to procure the drug, even with the help of donations. We expect at least the process of accessing the drug to get faster with approval,” said Archana Panda, the co-founder and director of patient advocacy for Gurgaon-based patient support organisation, CureSMA. 

ThePrint has reached Novartis over emails for details related to the drug’s India launch timeline and possible cost. This report will be updated if and when a reply is received.

Big buzz around the therapy

Over the last few years, the three drugs Spinraza, Zolgensma, and risdiplam—approved by the US Food and Drug Administration for Spinal Muscular Atrophy (SMA)—have considerably improved the quality of life for SMA patients.

There are three categories of Spinal Muscular Atrophy patients—SMA type 1, type 2, and type 3—with type 1 suffering from the most severe form of the disorder. The majority of patients with type 1 die in infancy.

Spinal Muscular Atrophy type 1 kids usually have more severe symptoms, noticeable within the first six months of life. Those with milder forms struggle with less severe symptoms, with a high possibility of them going unnoticed till the child is 18 months or older. 

The gene affected in Spinal Muscular Atrophy is the “survival of motor neuron” gene (SMN1 and SMN2). In 95 percent of SMA cases, both copies of the SMN1 gene are missing. 

All patients with Spinal Muscular Atrophy have several copies of the SMN2 gene. However, the SMN2 gene produces only a small amount of functional SMN protein—the more copies of the SMN2 gene a child has, the milder the disease.

Of the three drugs, Spinraza and risdiplam apply to all types of Spinal Muscular Atrophy, with patients requiring them lifelong. Zolgensma—developed by US-based AveXis and later acquired by Novartis—is a one-time gene therapy treatment that replaces the defective or missing gene, SMN1.

Global clinical trials of the drug showed that 95 percent of the babies with Spinal Muscular Atrophy type 1, given the drug before the onset of the disease at age two years, survived compared to just 31 percent surviving without the drug. 

The staggering results have pushed thousands of parents with babies born with severe Spinal Muscular Atrophy to scramble for the therapy launched in the US in 2020. However, affordability is a huge hurdle—at the time of the drug’s roll-out in the US, it was the costliest drug in the world. 

“While one drug for SMA-risdiplam is available in India and costs about 72 lakh per year, the fact that Zolegnama can change the life of SMA type 1 patients in just one administration is why so many parents try to access it, desperately,” Panda pointed out. 

The drug promises to cure the condition completely, provided that the newborn screening detects the disease, and if symptoms have not manifested. And, while it can not reverse the damage before the administration of the gene therapy, it can arrest disease progression—an added advantage. 

The therapy, however, can also cause severe liver damage, which can be life-threatening in a few patients, and all patients receiving the treatment need routine monitoring. Paediatric neurologists stress a careful assessment of affected babies to confirm they are ‘fit‘ for receiving the treatment. 

A huge demand for the therapy

Given the global demand for this high-end drug, Novartis began its Zolgensma global managed access programme, the first of its kind for a one-time gene therapy, in 2020. The programme provided the drug free of charge to eligible patients in countries where it was possible to make Zolgensma available, where it had not yet received approval, or where no access pathway existed.

Since 2020, nearly 300 children with spinal muscular atrophy from 40 countries, including at least 50 Indian kids, according to CureSMA, were treated free of charge after their selection in a lottery system. The initiative, however, was shut down in December 2023 for most countries, and in July last year, for Indian patients, as well.

In India, at least 10 parents of the SMA-affected kids have managed to raise funds for the treatment through donations or crowdfunding, while most others have been struggling.

And, if the exorbitant cost of the drug is a dampener for parents, it is the same with many doctors. 

“For a country like India, astronomically expensive drugs will remain out of reach for most, unless there are steps to make them affordable for those who need them,” said Dr Rekha Mittal, a paediatric neurologist, working at Rainbow Children’s Hospital in Delhi, routinely seeing SMA patients.

(Edited by Madhurita Goswami)