Larimar nagsumite ng unang module ng rolling BLA para sa nomlabofusp

Larimar ay nagsimula ng rolling BLA submission na naghahanap ng accelerated approval para sa nomlabofusp sa Friedreich’s ataxia, kung saan ang unang module ay naisumite na at ang buong submission ay inaasahang makukumpleto sa ikalawang kalahati ng 2026. Ang pagkakahanay sa FDA ay nakumpirma kasunod ng Type B pre-BLA meeting, kasama ang pagtanggap sa umiiral na open-label data package at FXN bilang isang bagong surrogate endpoint. Inaasahan din ng kumpanya na mag-dose ng unang pasyente sa global confirmatory Phase 3 study sa Q3 2026.

Mahahalagang fact

Na-file: Ago 4, 2026, 11:02 AM
Kaganapan: Naisumite ang aplikasyon
Direksyon: Positibo
Kumpanya: LRMRLarimar
Asset: nomlabofusp
Pinagmulan: SEC 8-K
Kumpiyansa: May source

Sipi mula sa source

EX-99.1 2 lrmr-ex99_1.htm EX-99.1 EX-99.1 Larimar Therapeutics Reports Second Quarter 2026 Financial and Business Update • Positive longer-term open label study data further demonstrated nomlabofusp's well-characterized safety profile, sustained increases in skin frataxin levels, and continued improvements in key clinical outcome measures • High patient and investigator enthusiasm continues with additional participants in the OL study dosed in July and several adults and adolescents in screening as of month-end • Rolling BLA submission seeking accelerated approval underway, with the first module submitted and completion expected 2H 2026 • Dosing of first patient in global confirmatory Phase 3 study expected Q3 2026 • $156.3 million in cash, cash equivalents and marketable securities

SEC 8-K