A toddler from Nashik, Maharashtra, received a free 16 crore life-saving injection for a rare genetic disorder from a company in the United States, making it the most expensive drug in history. According to news agency PTI, Shivraj Daware’s parents said their son is the first patient of Spinal Muscular Atrophy (SMA) from India to receive Zolgensma, a gene replacement therapy, through a lottery.
Shivraj received the injection, which doctors say is the most effective drug for SMA patients, ahead of his second birthday, according to PTI, citing Shivraj’s parents. Following the initial diagnosis, Shivraj was referred to Mumbai’s Hinduja Hospital. Dr. Brajesh Udani, a neurologist at Hinduja Hospital, told Vishal Daware that Zolgensma could save his son’s life, according to PTI. The huge sum was impossible to arrange, according to Vishal Daware, who owns a photocopy shop in Nashik.
Dr. Udani suggested that they apply for a lottery to be held by a US-based firm for conducting clinical trials, and that if they are lucky, they could get the injection for free. Shivraj was chosen by the company in a lucky draw on December 25, 2020, and received the injection on January 19, 2021, at the Hinduja Hospital.
“SMA 1 is a genetic disorder. One out of 10,000 children gets affected by the disorder. It slows down the movement of the child and the muscles stop working. Later, it leads to the death of the child,” Dr Ramant Patil, who treated Shivraj, before, was quoted as saying by PTI.
The Centre offered a two-year-old girl from Tamil Nadu a special exemption from paying customs duty and GST for importing the life-saving drug in July. Import taxes on 55ml of the drug Zolgensma have been exempted under exceptional circumstances and in the public interest, according to the Union finance ministry.
Previously, a Hyderabad-based couple raised Rs. 16 crore through crowd-funding in three-and-a-half months to purchase the injection for their three-year-old son with SMA.
Novartis announced last year that Zolgensma had received FDA approval in the United States. Zolgensma, according to the Swiss pharmaceutical company, is a one-time treatment for SMA, a disease that affects about 1 in 10,000 births and causes death or the need for permanent ventilation in 90% of cases by the age of two.
