Skip to records

Therapeutics

229Aging Biotech
100 of 229 records

Nomlabofusp

Source date not recordedAnalyse record ↗

Mitochondrial protein-replacement therapy in late-stage development for systemic frataxin deficiency in Friedreich's ataxia.

Modality
subcutaneous cell-penetrating recombinant human frataxin fusion protein
Availability (US)
Phase 3 registered as Recruiting and Phase 2 open-label extension Recruiting; rolling BLA submission underway
Biomarkers
tissue frataxin concentrations; modified Friedreich Ataxia Rating Scale; upright stability; safety and immunogenicity
Hallmark targeted
mitochondrial dysfunction, impaired iron-sulfur-cluster biology, loss of proteostasis
Kind
investigational biologic
Clinical trials
NCT07778836 — Phase 3, Recruiting on August 31, 2026 registry update; NCT06447025 — Phase 2 open-label extension, Recruiting on September 3, 2026 update; NCT06681766 — pediatric Phase 1, Terminated