Roche’s Evrysdi therapy for the treatment of children with type 1 spinal muscular atrophy (SMA) has been shown to increase chances of survival and sustain improvements in achieving key motor milestones, according to results from drug co-developer PTC Therapeutics’ FIREFISH trial.
The treatment was shown to improve head control, sitting, rolling over, and further developing towards acquiring the ability to stand and walk in infants.
Data from Part 2 of the trial on 41 patients demonstrated that at 24 months of treatment, 93% of infants were alive and 83% were alive without permanent ventilation. There were no new deaths between months 12 and 24.
Whereas infants with type 1 SMA older than 12 months generally require feeding support, 92% of patients treated with Evrysdi also maintained their ability to feed orally at month 24.
In addition, fewer hospitalisations were observed during the second year of treatment with Evrysdi compared with the natural course of the disease, with 34% not requiring hospitalisation during 24 months of treatment.
SMA is a severe, progressive neuromuscular disease that can be fatal. It affects approximately 1 in 10,000 babies and when untreated is the leading genetic cause of infant mortality. SMA is caused by a mutation of the survival motor neuron 1 gene, which leads to a deficiency of SMN protein.
Evrysdi is designed to treat SMA by increasing and sustaining the production of the SMN protein, which is found throughout the body and is critical for maintaining healthy motor neurons and movement.
Stuart W Peltz PhD, CEO of PTC Therapeutics, said: ”We’re encouraged to see these patients reach key motor and mobility milestones, which would never have been realised in the natural course of the disease.
”The long-term data presented today build upon the robust pool of evidence supporting Evrysdi as a safe, effective and convenient at-home treatment option for SMA patients globally.”
This is a syndicated feed from Pharmafile