Larimar Therapeutics Inc. (NASDAQ:LRMR) said Monday it has submitted the first module of a rolling Biologics License Application to the U.S. Food and Drug Administration seeking accelerated approval for nomlabofusp, its investigational treatment for Friedreich’s ataxia.
The filing follows feedback from a multidisciplinary Type B pre-BLA meeting in which the FDA indicated the existing data package appears capable of supporting a biologics application.
The clinical-stage biotechnology company also released updated long-term data from its ongoing open-label study of nomlabofusp in adolescent and adult patients with Friedreich’s ataxia, a rare, progressive neurological disease.
FDA Supports Rolling Submission Strategy
Larimar said the FDA agreed to a rolling BLA submission, with the remaining application modules expected to be submitted during the second half of 2026.
According to the company, the agency reaffirmed its willingness to consider frataxin, or FXN, as a novel surrogate endpoint.
The FDA also indicated that Larimar’s exposure-response analyses linking nomlabofusp exposure to clinical outcomes could support the application.
Additionally, the FDA noted that exploratory gene expression and lipid biomarker data may provide further evidence supporting both the proposed surrogate endpoint and nomlabofusp’s mechanism of action.
Long-Term Study Shows Durable Safety Profile
As of June 2026, 43 participants had received at least one dose of nomlabofusp in the ongoing open-label study, with 22 patients still enrolled. Some participants have remained on treatment for more than 800 days.
More than 10,000 doses have been administered to date. Larimar said daily dosing was generally well tolerated, with the most common adverse events consisting of mild to moderate injection-site reactions that declined over time and did not result in study withdrawals.
However, 10 of 41 participants experienced anaphylaxis, including nine individuals previously exposed to nomlabofusp. All affected participants recovered following standard treatment without lasting complications.
Overall, 21 participants discontinued the study, including eight withdrawals related to anaphylaxis or other adverse events.
Clinical Outcomes Compared Favorably With Natural History Data
Larimar reported directional improvements across multiple clinical measures, including modified Friedreich’s Ataxia Rating Scale (mFARS), activities of daily living scores, and the nine-hole peg test after one year of treatment.
Patients receiving nomlabofusp demonstrated a mean 1-point improvement in mFARS at one year, compared with a 1.6-point worsening observed in a matched reference population from the Friedreich’s Ataxia Clinical Outcome Measures Study, resulting in a 2.6-point difference.
At 18 months, treated patients showed a mean 2.3-point improvement in mFARS, versus a projected 2.3-point decline in the reference population.
Among ambulatory patients who completed one year of treatment, none progressed to non-ambulatory status, while one previously non-ambulatory patient became ambulatory.
Larimar plans to dose the first patient in its global confirmatory Phase 3 study in the third quarter of 2026 and is targeting a potential commercial launch in mid-2027, if approved.
LRMR Stock Price Activity: Larimar Therapeutics shares were down 15.16% at $3.10 at the time of publication on Monday, according to Benzinga Pro data.
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