New Medicines

TranslarnaDuchenne muscular dystrophy in non-ambulatory patients


Licence extension / variation
PTC Therapeutics
PTC Therapeutics

Development and Regulatory status

August 2020
Jun 20Extension of indication to specifically include non-ambulatory patients was refused by the CHMP of the EMA, who stated that "robust clinically relevant efficacy of ataluren in non-ambulant Duchenne muscular dystrophy (DMD) patients has not been demonstrated". However, amendment was approved to remove the statement "Efficacy has not been demonstrated in non-ambulatory patients" from section 4.1 of the SmPC [4]
Jun 20PTC Therapeutics announce that the EMA´s Committee for Medicinal Products for Human Use has recommended by a majority of votes to remove the statement "efficacy has not been demonstrated in non-ambulatory patients" from the SmPC for Translarna™ (ataluren). This label change enables healthcare professionals to use their clinical judgement to make treatment decisions for their patients on Translarna who have lost ambulation [3].
Oct 19EU second negative opinion recommending refusal of a licence change to add the treatment of patients with Duchenne muscular dystrophy who are no longer able to walk. It had issued its initial opinion on 27 June 2019. The company had provided data to show that the body handles the medicine in a similar way in patients who are able to walk and those who cannot. In addition, the company presented the results of a study involving 94 patients, 44 of whom were no longer able to walk. Although the main objective of the study was to assess the long-term safety of Translarna, the study also investigated the effectiveness of treatment in patients unable to walk, measuring changes in lung function. The company then compared the results with historical data from patients with Duchenne muscular dystrophy recorded in the database of the CINRG (Cooperative International Neuromuscular Research Group). The Agency concluded that the fact that Translarna is handled by the body in a similar way in patients who can walk and those who cannot was not enough to confirm the effectiveness of Translarna in these patients. The additional data from the study could also not confirm the benefit of Translarna in patients no longer able to walk because there were problems with the way data from the CINRG database, which was used to indirectly compare the effects of Translarna, were selected and analysed [2].


Dystrophin stimulant
DMD affects about 1 in 3,500 newborn males [1].
Duchenne muscular dystrophy in non-ambulatory patients

Trial or other data

Jun 20The proposal to the EMA to include non-ambulatory subjects with nmDMD in the indication for ataluren (Translarna) was claimed based on results of PTC-124-GD-019-DMD (study 019), a non-randomised, open-label, uncontrolled, multicentre, extension study evaluating primarily the tolerability and long-term safety of ataluren over approximately 240 weeks of use. 90 out of 96 subjects in study 007 rolled-over to study 019. 64 of these were non-ambulatory. The dose regime of ataluren was the same as that approved for the ambulatory patients aged >2 years of age. Secondary outcomes included spirometry variables and the EK in non-ambulatory patients, assessed at screening (visit 1), week 48 (visit 6), and week 48 through week 240 or premature discontinuation. 45 non-ambulatory patients had non-missing information on age at first symptom and age at percentpredicted FVC <60%. They formed the basis for the main analyses. The efficacy outcomes were compared to historical controls, derived from the CINRG-database. A propensity-score approach was made to adjust the comparisons for confounding [4]

Evidence based evaluations