LRMR submits initial Biologics License Application for rare disease therapy, targeting accelerated approval based on surrogate endpoint data.
Larimar Therapeutics submitted the first module of its Biologics License Application to the FDA for Nomlabofusp, seeking accelerated approval for Friedreich’s ataxia treatment. The company plans to file remaining modules in the second half of 2026.
The therapy showed positive results in an open-label study involving adult patients. The FDA confirmed existing data, based on skin frataxin as a surrogate endpoint, is sufficient for the application. Analysts at Baird lowered the stock’s price target from $7 to $5 but maintained an Outperform rating.
Shares of LRMR, trading under $10, reflect investor caution amid clinical benefits and safety concerns highlighted in recent updates.