Mar. 21 at 12:33 PM
$PLX Interim results are out from the BRIGHT F51 study (NCT03614234) which I kind of lost sight of. It is an ongoing open-label extension following patients switched from prior ERT to Elfabrio in the earlier BRIGHT study. This interim analysis covers 29 patients treated E4W for 3–5 years (cutoff December 31, 2022), and builds on the shorter-term data that likely supported the recent EU approval. The paper was published yesterday in Orphanet Journal of Rare Diseases.
https://link.springer.com/article/10.1186/s13023-026-04303-8
Since the EU approval is very recent and the paper was published afterwards, it did not directly influence the regulatory decision (which relied on prior data from BRIGHT and earlier extensions). However, it provides valuable additional long-term reassurance (up to 5 years) beyond what was available for the approval process. It will give clinicians and patients more confidence in E4W for stable patients and support decisions to switch. Should help with uptake where Elfabrio is already approved.
