A toddler from Nashik in Maharashtra has got a ₹16 crore life-saving injection, the most expensive drug in history, for a rare genetic disorder from a company in the US free of cost. The parents of Shivraj Daware said their son is the first patient of Spinal Muscular Atrophy (SMA) from India to get Zolgensma, a gene replacement therapy, through a lottery, according to news agency PTI.
PTI reported citing Shivraj’s parents that he got the injection, which doctors say is the most effective drug for SMA patients, ahead of this second birthday. Shivraj was referred to Mumbai’s Hinduja Hospital after the initial diagnosis. Vishal Daware told PTI that Hinduja Hospital neurologist Dr Brajesh Udani suggested Zolgensma could save his son’s life. Vishal Daware, who runs a photocopy shop in Nashik, said arranging the huge amount was impossible.
Vishal Daware said Dr Udani suggested they could apply for a lottery to be brought out by the US-based firm for conducting clinical trials and that they could get the injection free of cost if they are lucky. Shivraj was selected by the company in the lucky draw on December 25, 2020, and was administered the injection at the Hinduja Hospital on January 19, 2021.
“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.
In July, the Centre offered a special exemption from payment of customs duty and GST for importing the life-saving drug for a two-year-old girl from Tamil Nadu. The Union finance ministry said in a statement that import taxes on 55ml of drug Zolgensma have been exempted under exceptional circumstances and in the public interest.
Before that, Hyderabad-based couple mobilised ₹16 crore in three-and-a-half months through crowd-funding to buy the injection for their three-year-old son with SMA.
Last year, Novartis said it had received approval from US regulatory for Zolgensma. The Swiss pharmaceutical company said Zolgensma was a one-time treatment for SMA, a disease that affects about 1 in 10,000 births and which results in death or the need for permanent ventilation by the age of two in 90 per cent of cases.