Larimar completes rolling BLA for nomlabofusp in Friedreich's ataxia

Larimar Therapeutics announced on November 9, 2026 that it had completed a rolling Biologics License Application submission to the FDA seeking accelerated approval of nomlabofusp for Friedreich's ataxia.¹ Nomlabofusp is an investigational protein replacement therapy that aims to address frataxin deficiency, the underlying cause of FA, by delivering human frataxin to mitochondria.¹

The BLA relies on frataxin concentrations measured in skin as a potential surrogate endpoint, drawing on data from an ongoing long-term open-label study. The company said all evaluable participants reached one-year skin frataxin levels comparable to those seen in asymptomatic heterozygous carriers, with sustained increases over time.¹ At one year, several clinical outcome measures trended in a favorable direction compared with a natural history reference population that was expected to worsen.¹ Exploratory markers, including plasma lipids and peripheral tissue gene transcription, also showed directional improvement versus placebo-treated patients.¹

Regarding regulatory interactions, the FDA had indicated it would consider frataxin as a novel surrogate endpoint and said Larimar's analysis linking drug exposure to clinical outcomes was the kind of evidence that could support the submission.¹

Larimar is concurrently enrolling the Phase 3 "FORWARD-FA" trial, a double-blind, placebo-controlled study testing daily subcutaneous nomlabofusp over 18 months in ambulatory patients ages 2 to 40 who have not previously received the drug, with about two-thirds of participants expected to be under 21.¹

The company laid out a timeline: BLA acceptance is expected in early 2027, an open-label safety update in the first quarter of 2027, U.S. approval by mid-2027, potential award of a Rare Pediatric Disease Priority Review Voucher around the same time if approved, and a targeted launch in the third quarter of 2027 if approved.¹

Separately, in a related Item 2.02 disclosure, Larimar reported approximately $128.7 million in cash, cash equivalents and marketable securities as of September 30, 2026¹, though it noted its quarter-end financial close process was not yet complete and the figure could change.¹