Roche's oral therapy 'Evrysdi' to provide cost-effective alternative against fatal genetic disorder SMA in India

Global drug major Roche has launched 'Evrysdi' (Risdiplam) in India for the treatment of Spinal Muscular Atrophy (SMA). As per the company, Evrysdi is the first and only approved treatment available in the country against this fatal genetic disorder.

Until now, SMA in India was curable only with the two most expensive and imported gene therapy injections -- Spinraza and Zolgensma -- from Biogen-Ionis and Novartis Gene Therapies. Treatment for this genetic disorder costs over Rs 16-18 crore in India, which makes it the costliest therapy in the world.

SMA is a severe, progressive rare neuromuscular disease that can be fatal. It affects around one in 10,000 live births globally and one in 7,744 live births in India. It is the leading genetic cause of infant mortality. About 68% of Type I SMA patients die before their first birthday and 82% die before the 4th birthday, say estimates.

SMA is caused by a mutation of the survival motor neuron 1 (SMN1) gene, which leads to a deficiency of SMN protein. Evrysdi helps in increasing the production of SMN protein.

Sources said at present, a single dose of Spinraza injection costs Rs 87 lakh. Seven doses are given in the first year of the treatment, followed by three doses every year for the rest of the patient's life.

Zolgensma, a one-time therapy that is administered to patients less than two-year-old, costs about Rs 16 crore in India. Both of these drugs have not been launched in India, so they are imported.

Both these drugs recently made news as several patients in India got crowdfunding for treatment. Many state leaders were also seeking exemption of import duties on these two drugs.  

Evrysdi, a disease-modifying therapy, is the first and only approved treatment for SMA patients across all types. It is administered orally at home daily (it is supplied as a powder, which is constituted into a liquid solution and taken once daily by mouth or feeding tube if required).

One bottle costs about Rs 6 lakh, with a dosage of 0.75 mg/ml powder for oral solution.

Roche said it plans to offer the drug to patients in India at a much discounted price and under the patient support programme (PSP), among all age groups.

Under this, 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 each bottle purchased by the patient.

For an infant weighing 5kg, one bottle will last for 60 days. The infant will need 6 bottles a year. For a patient weighing more than 20 kg, a bottle lasts only for 12 days and he/she requires 31 bottles a year. "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," said V Simpson Emmanuel, CEO and Managing Director, Roche Pharma India.

"Given the majority of people with SMA in India remain untreated, we believe Evrysdi, with its highly efficacious clinical profile and oral administration advantage, will offer meaningful benefits for many living with this rare neurological disease," said Dr Bruno Jolain, chief medical officer, Roche Pharma India.

Administration of Evrysdi requires no hospitalisation, no anaesthesia, no specialised care centre, no complex administration and no steroids. A simple oral administration gives SMA patients, treating physicians and caregivers more control over their daily lives, he said.

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