Ambulatory Duchenne patients who are 5 years and older with a nonsense mutation can now access a treatment that targets the underlying cause
SOUTH PLAINFIELD, N.J., April 29, 2019 /PRNewswire/ — PTC Therapeutics, Inc. today announced that Translarna™ (ataluren) has been granted marketing approval from the Brazilian National Health Surveillance Agency (ANVISA) under rare diseases procedure, for the treatment of ambulatory children five years and older with Duchenne muscular dystrophy caused by a nonsense mutation. Patients now can have access to a treatment that targets the underlying cause.
“The regulatory approval from the Brazilian authorities will accelerate access to Translarna for the many patients who have been waiting for treatment,” stated Eric Pauwels, Senior Vice President and General Manager of the Americas of PTC Therapeutics, Inc. “We are committed to working quickly to make Translarna available to all patients in Brazil who may benefit.”
Primarily affecting males, Duchenne muscular dystrophy is a rare, irreversible, and fatal genetic disorder that results in progressive muscle weakness from early childhood and leads to premature death in the mid-twenties due to heart and respiratory failure. Patients with Duchenne can lose the ability to walk as early as age ten, followed by loss of the use of their arms. Duchenne patients subsequently experience life-threatening lung complications, requiring the need for ventilation support, and heart complications in their late teens and twenties.
“Muscle damage in Duchenne starts very young, so early diagnosis and treatment is critical to maintain muscle function and delay disease progression,” stated Dr. Alexandra Prufer, Associate Professor of Pediatric Neurology, Department of Pediatrics, Medical School, The Federal University of Rio de Janeiro. “Treatment measures should be started once children are diagnosed, when there is the most amount of muscle to effect.”
Ataluren, discovered and developed by PTC Therapeutics, Inc., is a protein restoration therapy designed to enable the formation of a functioning protein in patients with genetic disorders caused by a nonsense mutation. A nonsense mutation is an alteration in the genetic code that prematurely halts the synthesis of an essential protein. The resulting disorder is determined by which protein cannot be expressed in its entirety and is no longer functional, such as dystrophin in Duchenne muscular dystrophy. Translarna, tradename ataluren, is licensed in the European Economic Area for the treatment of nonsense mutation Duchenne muscular dystrophy in ambulatory patients aged two years and older. Ataluren is an investigational new drug in the United States.
About PTC Therapeutics, Inc.
PTC is a science-led, global biopharmaceutical company focused on the discovery, development and commercialization of clinically-differentiated medicines that provide benefits to patients with rare disorders. PTC’s ability to globally commercialize products is the foundation that drives investment in a robust pipeline of transformative medicines and our mission to provide access to best-in-class treatments for patients who have an unmet medical need.