BALA CYNWYD, PA — Larimar Therapeutics, Inc. (Nasdaq: LRMR) has announced an updated timeline for submitting its Biologics License Application (BLA) for nomlabofusp, a potential first-in-class treatment for Friedreich’s Ataxia (FA). The revised schedule follows written feedback from the U.S. Food and Drug Administration (FDA) under the Support for Clinical Trials Advancing Rare Disease Therapeutics (START) program, providing clear expectations for safety data inclusion.
The FDA has recommended that safety data encompass at least 30 participants with six months of continuous nomlabofusp exposure, including at least 10 participants with one-year exposure. Most data should derive from those receiving the 50 mg dose. Larimar now plans to submit its BLA seeking accelerated approval in the second quarter of 2026.
“We are thrilled to have clarity from FDA on the safety database recommendations following submission of safety information included in a briefing package from our nomlabofusp program,” said Carole Ben-Maimon, M.D., the company’s President and CEO. “Importantly, we now have written recommendations from FDA on critical elements of the BLA submission including the safety database as well as the use of skin frataxin (FXN) concentrations as a reasonably likely surrogate endpoint (RLSE). Enrollment in our open-label extension (OLE) study continues to progress and we have recently expanded enrollment to include adolescents and patients who have not participated in prior clinical studies and therefore have not been exposed to nomlabofusp previously. Based on the FDA’s safety database recommendations and our plan to request approval to treat a broad population of patients including adults and children, we now plan to submit our BLA seeking accelerated approval in the second quarter of 2026.”
The company’s OLE study, which evaluates the long-term safety, pharmacokinetics, and biomarkers of nomlabofusp through once-daily subcutaneous injections, continues to gather data. Thirty to forty participants, including adolescents, are expected to contribute to this next cohort, with results anticipated by September 2025.
“Our long-term OLE study is further advancing, with some participants now on treatment for up to 15 months. This includes exposure at both the 25 mg and 50 mg doses,” said Rusty Clayton, M.D., Chief Medical Officer of Larimar Therapeutics. “The high adherence rates we are seeing for daily subcutaneous injections in participants over the long term is very encouraging. We have begun transitioning adolescents from the PK run-in study and have amended the protocol to include patients who have never participated in any of our prior clinical trials. Overall, we are pleased with our progress and the recommendations we now have from FDA on the safety database to achieve our near-term registrational goals.”
Nomlabofusp is an investigational therapy focused on addressing FA by increasing frataxin levels in the body. The FDA is considering skin frataxin concentration as a surrogate endpoint for potential accelerated approval, with final acceptability to be determined during BLA review.
Larimar is also preparing a global Phase 3 study as a confirmatory trial, with sites identified in the United States, Europe, Canada, and other regions. The study will run concurrently with the expected BLA submission, aligning with regulatory goals to expand treatment access worldwide.
By integrating FDA guidance into its clinical development program, Larimar Therapeutics remains committed to advancing patient care and addressing significant unmet needs for individuals living with Friedreich’s Ataxia.
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