Mumbai: Nair hospital to launch new drug for spinal muscular atrophy

The launch will be one of the several community based initiatives Nair Hospital have undertaken as part of the celebrations, including the launch of genome sequencing for Covid-19

As part of its centenary celebrations, BYL Nair Hospital — one of the four medical colleges run by the Brihanmumbai Municipal Corporation (BMC) — will launch a new drug to treat spinal muscular atrophy (SMA), a genetic disease affecting the central nervous system. The launch will be one of the several community based initiatives Nair Hospital have undertaken as part of the celebrations, including the launch of genome sequencing for Covid-19.

Speaking to mid-day, Dr Ramesh Bharmal, dean of Nair Hospital, said, “It is for the first time that we are getting this injection in India. We are importing it from USA and it will be given to selected children. The disorder encompasses a group of inherited progressive, degenerative, neuromuscular disorders, because of which children die at a young age. It happens due to respiratory failure or they become wheelchair-bound for life,” said Dr Ramesh Bharmal, dean of Nair Hospital.

30 children registered

The drug, which has been imported from California, costs Rs 87 lakh per injection. The total annual cost of the therapy for a patient is nearly R6 crore in the first year and R3.2 crore in the subsequent years. The selected patients will receive the therapy lifelong. This rare genetic disease is said to affect one in 10,000 children and at present, 30 children have been screened and registered for the treatment at Nair Hospital.

“This genetic disorder affects the muscles of the body. The protein which is required for proper muscle function is absent or not generated,” explained Dr Alpana Kondekar, associate professor, Paediatric department and project coordinator.

She said till 2016, there was no treatment available for the disease, but now there are three.

“A gene therapy, which has to be given before two years of age and preferred in type 1 and type 2 of SMA, is a single-dose injection costing R16-R18 crore. The second is the intrathecal injection — the Spinraza — which is given in the spinal cavity. The third is an oral medication, which was recently launched in India and we will be giving it to two of our patients,” added Dr Kondekar.  

Direct Relief is a California-based non-profit NGO, which will provide the new drug Nusinersen (Spinraza) to Nair, for treatment of SMA under Spinraza Individual Patient Humanitarian Access Program (SIPHAP). An independent, international Medical Expert Committee (MEC) of SMA specialists has selected 17 most eligible candidates for Spinraza from Nair Hospital. Chief Minister Uddhav Thackeray will launch the drug on August 4.

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