BALA CYNWYD, PA — Larimar Therapeutics, Inc. has begun a rolling application for accelerated U.S. approval of its experimental Friedreich’s ataxia treatment, positioning the biotechnology company to potentially launch the therapy by mid-2027 if regulators determine the data support approval.
The clinical-stage drugmaker said it has submitted the first module of its Biologics License Application to the U.S. Food and Drug Administration for nomlabofusp after receiving feedback from a Type B pre-BLA meeting indicating that its existing data package appears capable of supporting a filing based on skin frataxin levels as a potential surrogate endpoint.
The remaining application modules are expected to be submitted during the second half of 2026.
The filing marks a key step for a company developing what could become the first disease-modifying therapy for Friedreich’s ataxia, a rare, progressive neurological disorder for which there are no approved treatments targeting the underlying cause of the disease.
Larimar also released updated data from its ongoing open-label study of daily injections of nomlabofusp in adolescent and adult patients.
Among nine participants treated for at least one year, all achieved and maintained skin frataxin levels above 50% of those seen in healthy volunteers, a threshold the company said is comparable to levels observed in asymptomatic carriers of the disease.
Clinical measures also showed signs of improvement compared with a matched population from the Friedreich’s Ataxia Clinical Outcome Measures Study, a long-running natural history study of the disease.
Patients treated with nomlabofusp for one year showed an average one-point improvement on the modified Friedreich’s Ataxia Rating Scale, while the FACOMS reference population worsened by an average of 1.6 points, according to the company.
At 18 months, treated patients improved by an average of 2.3 points compared with an estimated 2.3-point decline in the reference group.
The company also reported that one of six participants who were unable to walk at the start of treatment became ambulatory after one year of dosing, while none of the seven ambulatory participants lost that ability during the same period.
Safety remains a focus for investors and regulators.
Larimar said more than 10,000 doses of nomlabofusp have been administered in the study and that the treatment has generally been well tolerated. However, 10 of 43 participants experienced anaphylaxis, including one patient with no prior exposure to the drug.
All patients recovered after receiving standard treatment, the company said.
Chief Executive Officer Carole Ben-Maimon said the FDA’s feedback indicates that approval “will be a matter of review” and that the company is moving forward with both the regulatory filing and a confirmatory Phase 3 study.
Larimar expects to dose the first patient in its global Phase 3 trial during the third quarter and is targeting a commercial launch in mid-2027, contingent on regulatory approval.
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