Launched by Roche, Evrysdi is the first and only approved treatment in India for patients with spinal muscular atrophy.
Roche Evrysdi is an oral treatment for adults and children at least two months of age who suffer from muscle atrophy of the spine.
Roche has initiated oral therapy for adults and children suffering from spinal muscular atrophy (SMA). Named Evrysdi, it is the first and only treatment approved in India for SMA patients.
What is SMA?
Muscle atrophy of the spine (SMA) is an inherited disease that causes muscle weakness by gradually destroying motor neurons. These are nerve cells in the brain and spinal cord that allow a person to move, talk, swallow, and breathe by transmitting commands from the brain to the muscles that perform these functions.
Patients with SMA have a mutation in the survival motor neuron 1 (SMN1) gene, resulting in a deficiency of the SMN protein. This protein is essential because it controls muscles and movements and helps with nerve function.
August is dedicated to raising awareness of SMA.
SMA is typically diagnosed at about 18 months of age or in childhood and is the most common cause of infant death. It affects approximately every 10,000 live births worldwide and every 7,774 live births in India, making it the largest genetic cause of infant mortality.
There is currently no cure for SMA. However, studies to improve treatment are ongoing. Therapy and help are available to control symptoms. SMA patients have a normal life expectancy.
What is Evrysdi?
Evrysdi is a powder that is mixed with a liquid and given orally or through a feeding tube to people aged two months and older. It treats SMA by increasing the production of Survival Motor Neuron (SMN) protein.
After a global clinical trial was conducted among 450 volunteers, it was approved by the Indian health authorities 11 months after it received approval from the U.S. FDA. These experiments were performed in infants and adult volunteers up to 60 years of age. It also included patients who had previously received SMA treatment with other medicines.
Roche announced that it will offer three free bottles during the first two years of treatment for every two bottles a patient purchases to ensure that people can receive this treatment. From the third year, two bottles will be given free of charge for each bottle purchased by the patient.
- Known as Werdnig-Hoffmann’s disease or SMA in infants, SMA type I usually occurs before the age of six months. Without treatment, sick children never sit or stand and the majority of them usually die of respiratory failure before they are two years old.
- With children SMA type II symptoms usually occur at the age of 6-18 months. They can sit without support but are unable to stand or walk. Life expectancy is shortening, but they can live to youth or young adulthood.
- With children SMA type III (known as Kugelberg-Welander disease) symptoms are 18 months old and can walk independently. They may first have difficulty walking, jogging, climbing stairs, or getting up from a chair and may have trembling in their hands. They can live a normal life if they receive proper care.
- Individuals with SMA type IV they may experience mild to moderate proximal muscle weakness, among other symptoms, and may develop this disease after the age of 21 years.