Oral use of risperidone is extended to patients with spinal muscular atrophy

The US Food and Drug Administration (FDA) has approved an extension of the label for this drug to include patients with symptoms of spinal muscular atrophy.

Neuromuscular cells are affected in spinal muscular atrophy (SMA). Photo: shutterstock.

The indication for use of oral Risdylam—everyday, Genentech—approved by the U.S. Food and Drug Administration (FDA), has been extended to infants younger than two months of age, thanks to interim safety and construction data in a study currently underway, called RAINBOWFISH. This oral medication was first approved in 2020, based on guidelines for children over 2 years old, if they are patients with spinal muscular atrophy (SMA).

The study included 25 infants from birth to 6 weeks of age on a first dose, all of whom had this type of SMA, which was genetically diagnosed, but who still had no symptoms due to their young age.

Biotech company Genentech, in a press release, said that after 12 months of treatment with risdiplam, the majority of children with presymptomatic SMA have reached major motor milestones.

Of the six children with two or three copies of the SMN2 gene, all were able to sit after one year of active treatment, about two-thirds were able to stand, and the other half were able to walk independently; It was all children Alive at 12 months old Without permanent ventilation.

“The Evrysdi consent for children Infants without symptoms are particularly important, as treating SMA early, before symptoms start, can help infants reach motor milestones, Dr. Richard Finkel, lead investigator for the trial, said in the statement.

“By including SMA in newborn screening programmes, this approval provides the opportunity to begin treatment at home with Evrysdi once the diagnosis is confirmed,” added Finkel, director of the Experimental Neuroscience Program, St. Jude Children’s Research Hospital. , Memphis, Tennessee.

From newborns to the elderly?

The SMA is a genetic disease Rare and often fatal, it causes muscle weakness and progressive loss of movement.

Spinal muscular atrophy, which affects approximately 1 in 10,000 children, is caused by a mutation in the gene for motor neuron survival 1 (SMN1). The gene encodes the SMN protein, which is necessary for maintenance and motor neuron function.

Risdiplam small molecule It is administered orally, distributed centrally and peripherally which modulates splicing of pre-RNA From motor neuron survival 2 (SMN2) to increase SMN protein levels.

“We are proud of this achievement, which has the potential to make a real difference for people living with SMA and their caregivers,” Garraway added.

As part of the label extension, the prescribing information for risdiplam was also updated to include the two-year pooled data from Parts 1 and 2 of the FIREFISH study, which demonstrated long-term efficacy and safety in infants with symptomatic SMA type 1, the company indicated.

“Because of its efficacy in multiple settings, Evrysdi is now available for people with SMA, from asymptomatic newborns, to asymptomatic newborns,” said Levi Jarway, MD, chief medical officer of Genentech and head of global product development, in the statement. to the elderly.”

Source consult here.