Larimar nagsumite ng unang module ng rolling BLA para sa nomlabofusp sa Friedreich’s ataxia

Larimar ay nagsimula ng rolling BLA submission para sa accelerated approval ng nomlabofusp matapos kumpirmahin ng FDA na sapat ang kasalukuyang data package para sa review. Ang unang module ay naisumite; ang natitirang mga module ay inaasahan sa 2H 2026. Muling pinagtibay ng FDA ang kahandaang isaalang-alang ang skin FXN bilang isang bagong surrogate endpoint.

Mahahalagang fact

Na-file: Hun 29, 2026, 10:33 AM
Kaganapan: Naisumite ang aplikasyon
Direksyon: Positibo
Kumpanya: LRMRLarimar
Asset: nomlabofusp
Pinagmulan: SEC 8-K
Kumpiyansa: May source
FXN sa balat (1 taon): 100% (9/9) >50% ng katamtamang malusog na boluntaryo
Pagkakaiba ng mFARS (1 taon): pagkakaibang 2.6 puntos kumpara sa FACOMS
Antas ng anaphylaxis: 10/43 na pasyente

Sipi mula sa source

EX-99.1 2 lrmr-ex99_1.htm EX-99.1 EX-99.1 Larimar Therapeutics Reports Positive Open Label Data and Submission of First Module of Rolling BLA for Accelerated Approval of Nomlabofusp for Friedreich’s Ataxia • FDA alignment on submission of BLA data package in multi-disciplinary Type B pre-BLA meeting minutes; first module of rolling BLA submitted with remaining modules expected 2H 2026 • Daily nomlabofusp increased and sustained skin FXN levels at 1 year and 18 months; 100% (9/9) of participants achieved and maintained levels over 50% of mean levels in healthy volunteers (comparable to asymptomatic heterozygous carriers) at 1-year • Continued directional improvement across mFARS, FARS-ADL, 9-HPT, MFIS observed at 1 year of nomlabofusp treatment (n = 13) relative to a worsening in a FAC

SEC 8-K