world's most expensive drug Zolgensma
Zolgensma: What it is and why the cost
It costs Rs 18 cr a dose
This drug, which was first approved on May 24, 2019 by the US Food and Drug Administration, was approved by the United Kingdom's National Health Services (NHS) on March 9, 2020. It is manufactured by US-based Swiss bio-pharmaceutical company Novartis Gene Therapies.
"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 behind the scenes to ensure a price that is fair to taxpayers," he added.
Spinal Muscular Atrophy is a rare hereditary disease caused by one missing gene or the deficiency of a functional survival motor neuron 1 (SMN1) gene, according to Novartis. This results in rapid and irreversible loss of motor neurons, affecting main muscle functions like breathing, swallowing and basic movements. Since these neurons control muscle movement throughout the body, a lack of it could cause paralysis, severe muscle weakness and loss of movement. The prevalence of SMA is about 1 in 10,000 children. 1 in 54 people carry the genetic defect of SMA and two carriers have a 25% chance of having a child with SMA.
Zolgensma is the second and most effective drug for the disorder. The reason for its exorbitant cost is its miniscule market size in the drug manufacturing industry and its potential to save lives. "The disorder is rare and that is why we needed a highly specialized drug. The expertise required to make it and the research around it has taken very long. It's also a one-time dose; the fewer the cases the higher its price will be. Discovering and manufacturing a new drug can costs billions of dollars.
How will Zolgensma treat SMA?
According to the NHS, the drug contains a replica of the missing gene. The active ingredient onasemnogene abeparvovec passes into the nerves and restores the gene, which then produces proteins necessary for nerve function and controlling muscle movement. The dose is determined based on the weight of the patient. "This drug is a one-time intravenous infusion which is administered on a patient for over an hour and is used to treat children under two years of age," Desai said. A common side effect in patients is rise in liver enzymes, according to Desai. As per the safety guidelines of Novartis, those with pre-existing liver conditions are particularly at risk of acute liver injury and tests should be conducted to assess liver function prior to the treatment with Zolgensma.
