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.
Update 19 March 2026:
Larimar announced on 19 March that the FDA has granted them Breakthrough Therapy Designation for nomlabofusp for the treatment of adults and children with FA based on FDA’s review of available clinical data from open label study.
Larimar plans to publish open label study data from their phase 1/2 trial of nomlabofusp in Q2 2026.
Larimar plans to initiate screening for their global Phase 3 confirmatory study of nomlabofusp in Q2 2026, with dosing of the first patient expected to be in mid-2026.
They plan to submit a Biologics License Application (BLA) to the FDA for marketing authorisation of nomlabofusp in the US, seeking accelerated approval of nomlabofusp in the US, with submission of non-clinical and clinical modules in June 2026. Larimar say that the US launch of nomlabofusp is targeted for the first-half 2027, if the drug is approved in the US by then.
Read the press release here.
Update 14th May 2026:
In a press release on 14th May 2026, Larimar confirmed that the above mentioned plans were on track. There are ongoing plans for the BLA submission to the FDA, seeking accelerated approval of nomlabofusp in the US, with submission of non-clinical and clinical data in June 2026. If the drug is approved by the FDA then they are still targeting US launch of nomlabofusp in the first half of 2027. The plan remains to initiate dosing of the first patient in their Global Confirmatory Phase 3 Study in mid-2026, and to publish open label study data from their phase 1/2 trial of nomlabofusp in the second quarter of 2026.
Additionally, in April 2026 Larimar published data from a study in mice, rats, non-human primates and people with FA that support skin frataxin levels as a surrogate endpoint for nomlabofusp programme. You can read the article here.
You can read the press release here.
