PTC Announces Expansion of Translarna Label to Include Ambulatory Patients as Young as 2 Years Old in Brazil
PTC Therapeutics, Inc. announced that the Brazilian National Health Surveillance Agency, ANVISA (Agência Nacional de Vigilância Sanitária), has approved the expansion of the indication of Translarna (ataluren) to include ambulatory children aged 2 to 5 years old with nonsense mutation Duchenne muscular dystrophy (Duchenne).1 This broadens the use beyond the previous age range of 5 years and older.
“Expanding access to Translarna to children with nonsense mutation Duchenne in Brazil is important to us,” said Stuart W. Peltz, Ph.D., Chief Executive Officer of PTC Therapeutics. “We know that with Duchenne, early diagnosis and treatment gives children the best chance of preserving muscle function and delaying the progression of the disease.”
Duchenne is a severe progressive disease that leads to rapidly worsening muscle function with children often using a wheelchair by early adolescence and eventually requiring artificial ventilation to breathe. Translarna is the only treatment for the underlying cause of Duchenne caused by a nonsense mutation. Clinical trials and real-world evidence have demonstrated Translarna’s potential to slow disease progression and improve patient outcomes.