On 29th September 2025, the pharmaceutical company Larimar Therapeutics announced positive results from their phase II trial of the drug Nomlabofusp for FA.Â
Nomlabofusp is designed to increase levels of the frataxin protein which is reduced in people with FA.
So far, 65 people have been dosed with the drug in the trial, with 14 having taken it for over 6 months and 8 for over a year. Increases in frataxin to similar levels as carriers have been seen in 10 of the participants who took the drug for over 6 months.
Improvements across a number of clinical rating scales (including mFARS, FARS-ADL, 9-HPT and MFIS) have been seen in participants who took the drug in the shorter term and longer term when compared to people in the FACOMS natural history study who did not take the drug.
Larimar plans to submit a new drug application to the FDA in the second quarter of 2026.
Read more about the trial here.
Read their press release here.
