Genentech’s Evrysdi (risdiplam), a treatment for spinal muscular atrophy (SMA), has been given an extended approved by the US Food and Drug Administration (FDA) to include babies under two months old.
SMA is a chronic and progressive neuromuscular disease that affects around one in 10,000 babies, and it is the leading genetic cause of infant mortality. It is caused by a mutation of the survival motor neuron 1 (SMN1) gene that results in a deficiency of SMN protein. This protein is found throughout the body and contributes to a vital nerve function that control muscles and movement.
Nerve cells cannot function correctly without the SMN protein, and this can lead to muscle weakness over time. Depending on the form of SMA, the patient’s physical strength and their ability to walk, eat or breathe can be considerably compromised or lost.
The FDA’s extended approval was based on interim safety and efficacy data from the RAINBOWFISH trial, which evaluated the treatment in newborn babies. The study showed that after one year of treatment with Evrysdi 100% of babies with two or three copies of the SMN2 gene were able to sit up. The data also showed that 67% could stand, 50% of infants involved could walk independently and none needed permanent ventilation. There were no fatalities after 12 months of treatment.
With this approval, Evrysdi is now approved to treat SMA in children and adults of all ages and can be administered daily at home in liquid form either by mouth or via a feeding tube.
Levi Garraway, Genentech’s chief medical officer and head of global product development, said: “The priority review and subsequent approval of Evrysdi for babies under two months of age [addresses] the urgent ongoing need for additional treatment options for babies with SMA.”
“Because of its efficacy in multiple settings, Evrysdi is now available for people with SMA from pre-symptomatic newborns to older adults… [and] has the potential to make a real difference to those living with SMA and their caregivers.”
As part of the label extension, the Evrysdi prescribing information has also been changed to include recent two-year pooled data from parts 1 and 2 of the FIREFISH study, which shows long-term efficacy and safety in symptomatic infants diagnosed with type 1 SMA.
Evrysdi is approved in 81 countries worldwide and is under review in a further 27 countries.