Larimar Therapeutics submitted the first module of a rolling BLA for accelerated approval of nomlabofusp and reported positive long‑term open‑label data showing sustained skin FXN increases and clinical improvements.
Key Highlights:
- First module of rolling BLA for accelerated approval of nomlabofusp submitted after FDA Type B pre‑BLA alignment; remaining modules expected 2H 2026.
- Daily nomlabofusp raised and sustained skin FXN; 100% (9/9) of participants had ≥50% of mean healthy volunteer FXN at 1 year.
- Directional improvement in mFARS, FARS‑ADL, 9‑HPT and MFIS at 1 year (n=13) vs. worsening in FACOMS reference, with a 2.6‑point mFARS advantage at 1 year.
- Long‑term dosing generally well tolerated (>10,000 doses); 10 anaphylaxis cases among OL study participants, most with prior nomlabofusp exposure; all recovered.
- Dosing of first patient in global confirmatory Phase 3 expected Q3 2026; company targets mid‑2027 launch if approved.
Original SEC Filing:
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