Hyderabad boy battling rare disease gets Rs 16 cr worth medicine

Zolgensma is the world's most expensive drug and is currently not available in India. It is being imported from the USA at a cost of USD 2,125,000 (INR 16 crore). Spinal Muscular Atrophy is a progressive neuromuscular disease caused due to defect in the SMN1 gene

Hyderabad (Telangana): Three-year-old Ayaansh Gupta from Hyderabad, suffering from a rare disease Spinal Muscular Atrophy (SMA), has been administered with the world's most expensive medicine 'Zolgensma', after his parents mobilised Rs 16 crore through crowd-funding. Ayaansh, son of Yogesh Gupta and Rupal Gupta, was given Zolgensma on June 9 at Rainbow Children's Hospital, Secunderabad, under the supervision of Dr Ramesh Konanki, Consultant Pediatric Neurologist.

Zolgensma is the world's most expensive drug and is currently not available in India. It is being imported from the USA at a cost of USD 2,125,000 (INR. 16 Crores). Spinal Muscular Atrophy is a progressive neuromuscular disease caused due to defect in the SMN1 gene. The affected children develop muscle weakness involving upper and lower limbs initially, but over time develop breathing difficulty and swallowing difficulty. SMA affects one in 10,000 children generally, and there are nearly 800 children suffering from SMA in India currently, and thrice as many children die before they even reach their second birthday.

Ayaansh Gupta with his parents

Zolgensma is a single dose intravenous injection gene therapy in which the defective SMN1 gene is replaced through an adenoviral vector. Earlier, two children have been given Zolgensma at Rainbow Children's Hospital, Secunderabad, in August 2020 and April 2021, and the medicine was provided by Novartis free of cost, on compassionate grounds. Both these children are recovering quite well and making progress in their physical growth and development. Elaborating details, Dr Ramesh Konanki stated that currently, there are three proven treatments for children suffering from SMA. "They have to be given either of Zolgensma, Spinraza and Risdiplam. Unfortunately, none of these medicines is currently available in India, and all are hugely expensive.

Spinraza and Risdiplam need to be taken life-long, and costs around Rs 40-70 Lakh per year. Hundreds of children affected with SMA do not have access to any of these treatments due to the mammoth costs involved for treatment," Dr Ramesh said. The Consultant Pediatric Neurologist, on behalf of the medical community and the SMA community (CureSMA group), requested Central and state governments to negotiate with respective pharma companies and make these medicines available at an affordable price.

Ayaansh's parents' happiness knew no bounds as their son was administered the life-saving medicine at last. A visibly elated Yogesh Gupta said, "We are thankful to the team at Rainbow Children's Hospital, for taking good care of Ayaansh. Grateful to all the donors and ImpactGuru, who have given Ayaansh a gift of life by getting him the world's most expensive medicine. Please continue praying for Ayaansh's quick recovery. We would never have reached so far without the support of you all."

"The parents were able to arrange the amount after going through crowdfunding by ImpactGuru, and the union finance ministry has waived off the tax worth around 6 crores Rainbow Hospitals appreciate the perseverance and courage shown Ayaansh' parents for the relentless efforts to save their son's life. Also, I would like to request the Indian government to make this injection available in India for a price that can be afforded by every parent," said Dr. Preetham Kumar, Consultant - Pediatrician and Pediatric Intensive Care, Rainbow Children's Hospital Secunderabad.

Piyush Jain, Co-Founder, and CEO, ImpactGuru said, "It is heartening to witness the power of crowdfunding, a large number of donors that came together to support Ayaansh Gupta's family in their journey of saving their child. Ayaansh's ImpactGuru fundraiser raised Rs. 14.84 crore, cumulatively contributed from over 62,450 donors. The single highest donation received on the crowdfunding campaign in INR is 56 lakh and in USD is $7,000. We are super ecstatic at the moment as this is our third successful fundraiser for an SMA Type 1 case, in the last four months. Our SMA fundraisers have made history in India's medical crowdfunding industry," said Jain. "We hope more families come forward to utilize our healthcare financing platform in their time of need to get the best available treatment for critical illnesses," he added.

ANI