Swiss drug major Roche on July 29 launched Evrysdi (risdiplam), the first and only approved drug in India for Spinal Muscular Atrophy (SMA), a progressive rare neuromuscular disease.
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. 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 (SMN1) gene, which leads to a deficiency of SMN protein. This protein is found throughout the body and is essential to the function of nerves that control muscles and movement. Without it, nerve cells cannot function correctly,
leading to muscle weakness over time. 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.
Roche said since its launch, more than 4,000 SMA patients across 50 plus countries have benefitted from Evrysdi.
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One bottle of Evrysdi is priced at Rs 6 lakh in India. To further drive broader access to SMA patients in India, Roche announceed a patient support programme (PSP) for Evrysdi through which in the first two years of treatment, 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 one bottle bought by the patient.
Roche said it will provide free home delivery of Evrysdi to each and every patient.
The number of bottles or dosage is dependent on the weight and age of a person. For an infant weighing 5 Kg, one bottle will last for 60 days. This translates to about 6 bottles per year. A patient who is weighing more than 20 Kg requires about 31 bottles per year, which is the max use case. With PSP support, the patient would need to buy about 12 bottles per year in the first 2 years and about 10 bottles per year from the third year onwards.
“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."Today heralds a new journey of hope as we are all coming together to add colour into the lives of SMA patients in India with the launch of Evrysdi, the first and only approved treatment in India for patients living with SMA. This also marks our foray into rare disease treatment in India," he added.