Larimar Therapeutics shares key biomarker data, aims for accelerated approval for Friedreich’s ataxia drug

Lari­mar Ther­a­peu­tics an­nounced Mon­day that its Friedre­ich’s atax­ia treat­ment in­creased me­di­an lev­els of a key pro­tein called fratax­in in a mid-stage study.

The biotech is in talks with the FDA to use fratax­in bio­mark­er da­ta to sup­port a po­ten­tial ac­cel­er­at­ed ap­proval sub­mis­sion in 2025, though its treat­ment is still un­der par­tial clin­i­cal hold.

In the Phase II study, pa­tients with Friedre­ich’s atax­ia re­ceived ei­ther 25 mg or 50 mg of nom­labo­fusp or place­bo. Their skin cells were biop­sied and buc­cal cells (the cells in­side the cheek) were swabbed to mea­sure lev­els of fratax­in. When peo­ple lack fratax­in, a mi­to­chon­dr­i­al pro­tein, it re­sults in mus­cle weak­ness and a loss of bal­ance and co­or­di­na­tion, among oth­er symp­toms.

Unlock this article instantly by becoming a free subscriber.

You’ll get access to free articles each month, plus you can customize what newsletters get delivered to your inbox each week, including breaking news.