Ataluren Delays Duchenne Muscular Dystrophy Progression 

The STRIDE study (NCT04868656) data of heterozygous (XY) individuals with Duchenne muscular dystrophy (DMD) who were treated with ataluren (Translarna; PTC Therapeutics, South Plainfield, NJ) had slowed disease progression. Treatment with ataluren delayed loss of walking by more than 5 years in those treated with ataluren compared with standard of care (SoC) alone. Results also show pulmonary function decline was delayed by 1.8 years in those treated with ataluren vs SoC.

An analysis of a 5 years of registry data showed that participants treated with ataluren plus SoC lost ambulation at a median age of 17.9 years compared with 12.5 years for those on SoC alone. At 12 years, 80% of participants receiving ataluren plus SoC are still walking, compared with 52% of participants receiving SoC alone. Forced vital capacity (FVC) of 60% or less, the value at which respiratory therapy is typically required, occurred at a median age of 17.6 years with ataluren  compared with 15.8 years without ataluren.

"The 5-year analysis of the STRIDE registry clearly demonstrates Translarna's profound impact on changing the course of disease progression, said Stuart W. Peltz, PhD, chief executive officer of PTC Therapeutics. "We are all proud to see Translana's life-changing effect on (those) with Duchenne. The results robustness showing both a 5-year delay in loss of walking as well as its ability to prolong lung function confirms what we have seen in our clinical trials. These results add to the totality of the evidence of Translarna's benefit for the patients and their families."

The study enrolled 241 participants from the STRIDE patient registry over the past 5 years. The data was compared with natural history from a propensity-score matched cohort in a long-term natural history study (the Cooperative International Neuromuscular Research Group (CINRG) natural history database of DMD patients).