Swiss pharma company Roche has received an approval from the European Commission (EC) for its spinal muscular atrophy (SMA) drug Evrysdi.
The EC has approved Evrysdi (risdiplam) for the treatment of SMA in patients aged two months or older who have a clinical diagnosis of SMA type 1, type 2, type 3 or have up to four survival motor neuron 2 (SMN2) copies.
Evrysdi is administered daily at home in liquid form by mouth or by feeding tube. The SMN2 splicing modifier is designed to treated SMA caused by mutations in chromosome 5q that causes SMN protein deficiency.
The approval is based on data from two clinical studies – FIREFISH in symptomatic infants aged two to seven months and SUNFISH in children and adults aged two to 25 years old.
In the FIREFISH study, 41% of infants receiving Evrysdi achieved the ability to sit without support for at least five seconds, measured by the Bayley Scales of Infant and Toddler Development Third Edition (BSID-III).
In addition to improving motor function scores, 90% of infants treated with Roche’s drug were living without permanent ventilation at 12 months of treatment, and reached 15 months of age or older.
Positive results were also observed in the SUNFISH study, wherein children and adults treated with Evrysdi experienced a clinically meaningful and statistically significant improvement in motor function at 12 months compared to placebo.
SMA is a muscle-wasting disease that affects around one in 6,000 to 10,000 live births and is a leading cause of genetic death in infants.
Evrysdi becomes the third drug approved to treat the condition, following Biogen’s Spinraza (nusinersen) and Novartis’ Zolgensma (onasemnogene abeparvovec), and is the first that can be administered orally.
“Today’s approval of Evrysdi, the first and only SMA treatment with proven efficacy that can be taken at home, potentially transforms treatment options for a broad range of people with SMA living in the EU,” said Levi Garraway, chief medical officer and head of global product development at Roche.
“By avoiding the need for in-hospital administration, Evrysdi can reduce the treatment burden on those living with SMA, their caregivers and healthcare systems,” he added.
In a statement, Roche said it is working closely with reimbursement and assessment bodies in European countries to enable patient access to Evrysdi.
The SMA therapy is set to be accessible to patients in Germany ‘in the coming days’ and in France from early April.