The United States Food and Drug Administration has approved Evrysdi (risdiplam) for the treatment of Spinal Muscular Atrophy (SMA) in adults and children of two months and older. The drug has been approved for Type 1, 2, and 3 SMA.
The drug is approved based on two clinical trials. FIREFISH included infants aged 2 to 7 months, and SUNFISH included patients aged 2 to 25 years. SUNFISH included adults with Type 2 and Type 3 SMA.
In the FIREFISH study, 41% of patients were able to sit without support for at least 5 seconds as measured by the Bayley Scales of Infant and Toddler Development Third Edition (BSID-III). 90% of patients were alive after a year without ventilation reached 15 months or older.
In SUNFISH study, the drug has demonstrated clinically meaningful and statistically-significant improvement after 12 months of treatment compared to placebo.
The drug has shown safety profile across FIREFRESH and SUNFISH trials. Fever, diarrhea, and last were the frequent adverse events reported.
Spinal Muscular Atrophy is a rare genetic disease that causes weakness because patients lose motors neurons, which are involved in controlling the movement.
Evrysdi (risdiplam) is the first and only oral medication that can be administered at home. The drug can be administered daily at home by mouth or by a feeding tube, including scoliosis or joint contractures. The drug acts by increasing the production of the survival of motor neuron protein, a protein crucial for the health of motor neurons and movement. Genentech has announced that the drug will be available to patients through Accredo Health Group.
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