Larimar Therapeutics submits first BLA module for nomlabofusp in Friedreich's ataxia
Larimar Therapeutics said it has submitted the first module of a rolling Biologics License Application to the FDA for accelerated approval of nomlabofusp, after FDA meeting minutes said the existing data package appears sufficient for submission. The company also reported longer-term open label data, which showed improvements in some clinical measures alongside anaphylaxis in 10 of 43 patients, a safety finding shareholders should weigh.
Key points
- First module of the rolling BLA submitted; remaining modules expected in the second half of 2026.
- FDA meeting minutes say the existing data package appears sufficient for a BLA seeking accelerated approval; approval will be a matter of review.
- Anaphylaxis occurred in 10 of 43 patients in the open label study, 9 of whom had prior exposure to nomlabofusp.
- At 1 year, 100% (9/9) of participants had skin FXN levels above 50% of healthy volunteer mean; first patient dosing in a global Phase 3 study is expected in Q3 2026.
The filing
Written by AI on Nov 9, 2026, 10:08 AM ET, from this filing only. Written later: this filing was briefed after the fact, when past filings were added to the site, so the brief may be read with hindsight; the filing itself is the record of what was known on June 29, 2026.
Briefs are written by AI from the filing text and can contain mistakes — open the filing before acting on anything. Impact (1-5) and tone are the AI's reading of the filing for shareholders, not a price forecast. Not investment advice.