Swiss pharma company Roche has bagged an approval from the US Food and Drug Administration for its spinal muscular atrophy oral drug Evrysdi (risdiplam).
The drug has been approved for the treatment of SMA in adults and children aged two months or older. Evrysdi demonstrated clinically meaningful improvements in motor function across two clinical trials in people with different ages and levels of disease severity, including type 1, 2 and 3 SMA.
Infants achieved the ability to sit without support for at least five seconds, a key motor milestone not typically seen in the natural course of the disease The drug also improved survival without permanent ventilation at 12 and 23 months, compared to natural history.
Evrysdi is currently being studied in over 450 people as part of a large clinical trial programme in SMA, which includes infants aged 2 months to adults aged 60 with varying symptoms and motor function.
The FDA approval is based on data from two clinical studies designed to represent a broad spectrum of people living with SMA: FIREFISH in symptomatic infants aged two to seven months and SUNFISH in children and adults aged two to 25 years.
In the FIREFISH study, 41% of infants treated with Evrysdi achieved the ability to sit without support for at least five seconds as measured by the Bayley Scales of Infant and Toddler Development Third Edition (BSID-III). In addition, 90% of infants were alive without permanent ventilation at 12 months of treatment and reached 15 months of age or older.
In SUNFISH, patients treated with Roche’s drug experienced a clinically-meaningful and statistically significant improvement in motor function at 12 months compared to placebo.
“Given the majority of people with SMA in the U.S. remain untreated, we believe Evrysdi, with its favorable clinical profile and oral administration, may offer meaningful benefits for many living with this rare neurological disease,” said Levi Garraway, Chief Medical Officer and Head of Global Product Development at Roche.
“The strength and resolve of the SMA community has continually inspired us as we developed this first-of-its-kind medicine for SMA, so today we celebrate our collective accomplishment together with them,” he added.