PTCT
withdrew the EMA application for Translarna in cystic fibrosis
The CHMP had concerns and was of the provisional opinion that Translarna could not have been approved for the treatment of cystic fibrosis caused by a nonsense mutation. The main study failed to show that Translarna was more effective than placebo at improving lung function or reducing flare -ups in the overall study population. The company wanted to restrict use of Translarna to patients not using the inhaled antibiotic tobramycin to treat infections, since analysis of the data suggested that this antibiotic might reduce the effectiveness of Translarna. The HMP was however concerned that the modest benefit seen in patients not using tobramycin might be a chance finding, and further data from an ongoing study would be needed to confirm this effect.
The CHMP was also concerned about harmful effects on the kidneys seen in patients treated with Translarna. These side effects appeared to be due to the fact that Translarna may worsen the effects on the kidney of certain antibiotics given by injection (aminoglycosides and vancomycin ), which are important in treating infections in patients with cystic fibrosis.
The Committee had requested additional information to demonstrate that these side effects could be reduced by temporarily stopping treatment with Translarna
when these antibiotics are given. Therefore, at the time of the withdrawal, the CHMP was of the opinion that the benefits of Translarna in the treatment of cystic fibrosis did not outweigh its risks
There are no consequences on the use of Translarna in its authorised indication (Duchene)
«Después de nada, o después de todo/ supe que todo no era más que nada.»