New Treatment for Spinal Muscular Atrophy Discovered

Roche has launched an oral treatment for adults and children suffering from Spinal Muscular Atrophy (SMA). It is the first and only treatment approved in India for SMA patients.

Major Swiss drug company Roche launched Evrysdi (risdiplam), the first and only approved drug in India for Spinal Muscular Atrophy (SMA).

Evrysdi, a breakthrough orally administered treatment for SMA patients, was first approved by the USFDA in August 2020 and is made available in India within 11 months of US approval.

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‘Evrysdi, the only approved drug to treat Spinal Muscular Atrophy is available in India.’

The treatment cost varies depending on weight and age, but a patient above 20 kg would have to pay a maximum of Rs 72 lakh for a full year treatment during the first two years with the patient support programme of Roche.

SMA is a severe, progressive rare neuromuscular disease that can be fatal. It affects approximately one in 10,000 live births globally and one in 7744 live births in India and is the leading genetic cause of infant mortality.

SMA is caused by a mutation of the survival motor neuron 1 protein, which is found throughout the body and essential to the function of nerves that control muscles and movement. The deficiency of SMN protein causes improper nerve cells function, leading to muscle weakness over time.

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Depending on the type of SMA, an individual’s physical strength and their ability to walk, eat or breathe can be significantly diminished or lost.

More than 4,000 SMA patients across 50 plus countries have benefitted from Evrysdi. One bottle of Evrysdi is priced at Rs 6 lakh in India. To further drive broader access to SMA patients in India, Roche announced a patient support programme (PSP) for Evrysdi through the first two years of treatment.

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“We are betting big on solving complex challenges related to rare diseases as we believe no patient should be deprived an opportunity to live a healthy life, however complex or rare the disease is," said V Simpson Emmanuel, CEO and Managing Director, Roche Pharma India.

Roche will provide three bottles free for every two bottles bought by the patient. From the third year onwards, Roche will provide two bottles free for every bottle the patient bought.

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Source-Medindia