BALA CYNWYD, PA — Larimar Therapeutics Inc. (Nasdaq: LRMR) said the U.S. Food and Drug Administration has granted Breakthrough Therapy Designation to nomlabofusp for the treatment of adults and children with Friedreich’s ataxia, based on preliminary clinical data from an ongoing open-label study.
The clinical-stage biotechnology company said the designation and recent written feedback from the FDA followed a Support for Clinical Trials Advancing Rare Disease Therapeutics meeting and support its plan to submit a Biologics License Application in June 2026 seeking accelerated approval.
Breakthrough Therapy Designation is intended to expedite development and review of drugs for serious conditions when preliminary clinical evidence indicates the potential for substantial improvement over existing therapies.
Larimar said the designation was supported by data showing increases in frataxin protein levels in skin tissue and consistent directional improvement across four clinical measures after one year of treatment, including the modified Friedreich Ataxia Rating Scale, Activities of Daily Living score, 9-Hole Peg Test and Modified Fatigue Impact Scale.
Friedreich’s ataxia is a rare, inherited disease affecting an estimated 5,000 people in the United States and is characterized by progressive neurological impairment.
“There continues to be a substantial burden of disease affecting the estimated 5,000 children and adults in the U.S. living with FA,” Rusty Clayton, chief medical officer of Larimar, said in a statement. He said preliminary data reviewed by the FDA showed improvements in clinical measures in the context of increased tissue frataxin levels.
The company said the FDA reaffirmed its willingness to consider skin frataxin levels as a novel surrogate endpoint reasonably likely to predict clinical benefit in support of an accelerated approval application. The agency also confirmed that a matched reference population from the FACOMS natural history database could be used for comparisons of clinical outcomes, according to the company.
Larimar said the adequacy of the safety dataset will be reviewed at the time of BLA submission.
Carole Ben-Maimon, president and chief executive officer of Larimar, said the company remains on track to submit the BLA in June 2026 and plans to initiate a global confirmatory Phase 3 study in the second quarter of 2026.
Topline data from the ongoing open-label study intended to support the BLA submission are expected in the second quarter of 2026, the company said. If approved, Larimar is targeting a U.S. launch in the first half of 2027.
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