Roche Pharma India which in July 2021 forayed into rare disease treatment in India with the introduction of its Evrysdi drug for (Spinal muscular atrophy) SMA patients, said it has received encouraging response from the family of patients and support groups.
Evrysdi is expensive in the sense it costs ₹6 lakh a bottle. For a patient who is weighing more than 20 kg, one bottle will last for 12 days. This translates to about 31 bottles a year in the maximum use case.
With Roche’s patients support programme, the patient’s family would need to pay for about 12 bottles [₹72 lakh] a year in the first 2 years and about 10 bottles a year [₹60 lakh] from the third year onwards.
For an infant weighing 5 kgs one bottle will last for 60 days. This translates to about 6 bottles a year.
SMA is a group of hereditary diseases that progressively destroys motor neurons, the nerve cells in the brain stem and spinal cord that control essential skeletal muscle activity such as speaking, walking, breathing, and swallowing, leading to muscle weakness and atrophy.
Roche said Evrysdi is the first and only approved treatment available in India for SMA patients across all types.
V. Simpson Emmanuel, CEO and Managing Director, Roche Pharma India said “With Evrysdi, we aim to bring remarkable change in the standard of care and treatment for SMA patients in India.”
“The introduction of Evrysdi showcases our commitment to living its purpose of doing what patients need next. Considering the huge unmet need of SMA patients in India, we worked with the patient community, HCP community, and the regulators to fast track the approval process,” he said.
“Evrysdi is now synonyms to hope and bright future for many patients living with SMA. This also marks our foray into rare disease treatment in India,” he added.
Dr. M. Ranjith Kumar, Paediatric Neurologist, SRMC, Chennai in a statement said “SMA critically affects the quality of life of patients and is a leading genetic cause of death in infants. With Evrysdi now being available in India, it signals a bright future for the entire SMA patient community, across all clinical types and age groups.”
“Early initiation of therapy can significantly improve treatment outcomes, especially for infants. Moreover, Evrysdi can be orally administered, thereby enhancing treatment adherence and ease of administration. The availability of Evrysdi in India is a big boon for pediatric neurologists all over the country,” he added.
SMA affects approximately one in 10,000 live births globally and one in 7,744 live births in India. Without treatment, babies with the most severe form of SMA, Type 1, cannot sit unsupported.
As per data made available by Roche, 68% die by the time they complete two years and 82% before they celebrate their fourth birthday.
The primary cause of SMA is the defect in both copies of SMN1 gene in the patients.
Since SMA leads to progressive muscle weakness and loss of motor neurons, timely initiation of therapy is crucial.
Till 2016 there was no treatment option available for SMA. However, the development of a few novel therapies have provided relief for SMA patients around the globe.
For several years, Zolgensma was the only imported gene therapy for SMA available to Indian patients. Reportedly the cost is about ₹16-₹18 crore making it difficult for families to meet the expenses.
Roche said the administration of Evrysdi requires no hospitalisation, no anaesthesia, no specialised care centre, no complex administration and no steroids.
It said over 70 people with SMA in India have been supported by it through its Compassionate Use Programme (CUP) where the patients have received the product free of cost.
Evrysdi could be available through staggered payment and the treatment cost is bit lower due to Roche’s scheme, but it is still too expensive for patients and their families to afford.
A government sponsored programme or provision for CSR funding by corporates for such expensive treatment can provide big relief to such patients and their families.
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