Larimar Therapeutics Gains Breakthrough Therapy Designation and Raises $107.6M for BLA Pathway
Larimar Therapeutics secured Breakthrough Therapy Designation for nomlabofusp in Friedreich’s ataxia and plans to submit a BLA seeking accelerated approval in June 2026, targeting U.S. launch H1 2027. The company raised $107.6 million in February and holds $244.5 million pro forma cash, funding operations into Q2 2027.
1. Breakthrough Therapy Designation and FDA Alignment
In February Larimar was granted Breakthrough Therapy Designation for nomlabofusp in adults and children with Friedreich’s ataxia and received FDA agreement on using skin frataxin as a novel surrogate endpoint, selecting a reference population from the FACOMS database, and the Upright Stability Score as a primary endpoint for its confirmatory Phase 3 trial.
2. Development Milestones and Timelines
Larimar expects to report topline open-label study data in Q2 2026 to support its BLA, initiate global Phase 3 screening in Q2 2026 with first patient dosing by mid-2026, submit its BLA seeking accelerated approval in June 2026, and target a U.S. launch in the first half of 2027 if approved.
3. Financial Position and Cash Runway
The company closed a $107.6 million public offering in February 2026, resulting in $244.5 million in pro forma cash, cash equivalents and marketable securities as of December 31, 2025, which provides funding into the second quarter of 2027.