Larimar Submits First Module Of BLA For Nomlabofusp, Reports Positive Long-Term FA Data

Larimar Therapeutics

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(RTTNews) - Larimar Therapeutics, Inc. (LRMR) on Monday said it has submitted the first module of its rolling Biologics License Application (BLA) to the Food and Drug Administration seeking accelerated approval of nomlabofusp for the treatment of Friedreich's ataxia (FA), a rare, progressive neurological disease.

"In the Type B multi-disciplinary pre-BLA meeting minutes, FDA confirmed that our existing data package appears to be sufficient to support a BLA submission seeking accelerated approval based on skin frataxin as a potential novel surrogate endpoint and approval will be a matter of review," said Carole Ben-Maimon, President and Chief Executive Officer of Larimar.

The company expects to submit the remaining BLA modules in the second half of 2026. Larimar also reported positive data from an ongoing long-term open-label study of nomlabofusp in adolescent and adult FA patients. Daily treatment increased and sustained frataxin (FXN) levels in the skin, with all evaluable participants achieving and maintaining levels above 50% of the average seen in healthy volunteers at one year and 18 months.

The company said patients showed continued directional improvement across multiple disease measures after one year of treatment compared with a worsening seen in a natural-history reference population. One of six non-ambulatory patients at baseline became ambulatory after one year of treatment, while none of the seven ambulatory patients lost the ability to walk.

Nomlabofusp continued to demonstrate a generally well-tolerated safety profile during long-term treatment. Larimar reported anaphylaxis in 10 of 43 patients, with nine of those patients having been exposed to the therapy in a prior study.

The company expects to dose the first patient in its global confirmatory Phase 3 trial in the third quarter of 2026.

The stock is down more than 15% in pre-market trading after closing at $3.66 on Friday.