BioCentury
ARTICLE | Regulation

Seeking surrogates

Dystrophin remains top DMD surrogate endpoint, but other metrics on the rise

October 13, 2017 7:11 PM UTC

Thanks to flaws in PTC Therapeutics Inc.’s trials, the recent FDA advisory committee meeting to discuss Translarna ataluren did nothing to clarify what level of dystrophin expression would be necessary to support an accelerated approval for Duchenne muscular dystrophy.

The chance to determine what level of dystrophin expression will correlate with improvements in function will likely come in 2019, when Sarepta Therapeutics Inc. reports data from two Phase III studies for three of its exon-skipping compounds...