PTC Therapeutics has released data from phase II of their FIREFISH trial of a treatment called Evrysdi (risdiplam) in infants with type 1 spinal muscular atrophy (SMA.)
SMA is a disorder which becomes evident around six months of age, resulting in very floppy, weak arms and legs, as well as difficulty swallowing and breathing. Infants with the disorder typically cannot sit upright without support, and the majority of the afflicted infants do not survive beyond the first few years of their life.
The second phase of the FIREFISH study involved a pivotal, single-armed clinical protocol in which 41 infants with type 1 SMA received the treatment for two years, followed by an open label extension. The treatment itself is a survival motor neuron 2-directed RNA splicing modifier, which in lay terms means the drug targets the chromosomal mutations that cause SMA.
The results indicate that after two years of treatment, 93% of the infants were alive, and 83% did not require permanent ventilation. No new deaths were recorded between 12 and 24 months, and 92% of the infants were still able to feed orally after two years. A maintenance of the ability to swallow was recorded in 95% of the infants.
Further, there were fewer hospitalizations required than in the natural course of the disease. The primary endpoint of this phase was that the majority of infants would be able to sit without needing support for a minimum of five seconds, after the first year of treatment.
The treatment was approved by the FDA in treating adults with SMA, as well as children aged two months and older.
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