A decade after Translarna's initial marketing authorization in Europe, it looks like the drug's run in the region is coming to an end.
EMA’s human medicines committee (CHMP) has recommended not renewing the marketing authorisation for Translarna (ataluren), a medicine for treating patients with Duchenne muscular dystrophy whose disease is caused by a type of genetic defect called a ‘nonsense mutation’ in the dystrophin gene and who are able to walk.
Despite high list prices, England's National Institute for Health and Care Excellence (NICE) has recommended rare disease treatments from PTC Therapeutics and Alnylam Pharmaceuticals.
SOUTH PLAINFIELD, N.J., Oct. 11, 2022 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) announced today that recent clinical trial and real-world evidence on the efficacy and safety of Translarna™ (ataluren) in patients with nonsense mutation Duchenne muscular dystrophy (nmDMD) will be presented at the 27th International Annual Congress of the World Muscle Society (WMS) in Halifax, Nova Scotia, Canada.
In draft guidance published on Friday, it emerged NICE does not recommend PTC Therapeutics’ Translarna (ataluren) for the treatment of Duchenne muscular dystrophy (DMD) caused by a nonsense mutation.
SOUTH PLAINFIELD, N.J., June 20, 2022 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) will host a conference call Tuesday, June 21st at 8:00 a.m. E.T. to review topline results from Study 041 of Translarna™ (ataluren) in patients with nonsense mutation Duchenne muscular dystrophy. The call will be accompanied by a slide presentation which can be accessed through our online webcast.
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). This broadens the use beyond the previous age range of 5 years and older.
SOUTH PLAINFIELD, N.J., Oct. 25, 2021 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) today 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.
SOUTH PLAINFIELD, N.J., Sept. 20, 2021 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) today presented real-world results from the STRIDE patient registry demonstrating that treatment with Translarna™ (ataluren) delays loss of ambulation by more than five years in boys with nonsense mutation Duchenne muscular dystrophy (nmDMD) compared to standard of care (SoC) alone. Pulmonary function decline was also delayed by 1.8 years in those treated with Translarna and SoC.