ICER's final report gives DMD drug manufacturers no let up

The USA's Institute for Clinical and Economic Review (ICER) could scarcely have been more damning of two companies behind treatments for Duchenne muscular dystrophy (DMD), the most common fatal genetic disease diagnosed in childhood.

The ICER was critical of the companies in its earlier evidence report, published last month, and the failure of the firms to engage with the subsequent public meeting may have done their cause further harm.

The USA-based cost-effectiveness watchdog was assessing the comparative clinical effectiveness and value of two exon-skipping therapies, Exondys 51 (eteplirsen) and golodirsen, from Sarepta Therapeutics (Nasdaq: SRPT).