Larimar files complete US application for Friedreich's ataxia drug, aiming for 2027 launch

Larimar Therapeutics has finished submitting its marketing application for nomlabofusp, its Friedreich's ataxia candidate, to the US Food and Drug Administration. The company announced the milestone on 9 November 2026. It sets out a path to market in the second half of 2027, if the agency agrees.

Larimar expects the FDA to decide on whether to accept the filing for review in early 2027. It is aiming for approval in mid-2027 and a commercial launch in the third quarter of that year. These are company targets, not commitments from the regulator. The announcement gave no information on pricing, revenue expectations or launch preparations.

The application seeks accelerated approval and relies on frataxin levels measured in skin as a possible surrogate endpoint. Larimar describes it as a potentially novel measure, and the supporting data come from open-label studies, in which patients and investigators know who is receiving treatment. Whether the FDA treats skin frataxin as a credible predictor of clinical benefit is therefore the central question for the filing.

Larimar's Phase 3 FORWARD-FA study, intended to confirm the drug's benefit, is still enrolling patients. Context: accelerated approval lets the FDA clear medicines for serious conditions on the basis of a surrogate endpoint considered reasonably likely to predict clinical benefit, with confirmatory trials required after approval.