pp-156 Solution for injection
Dossier type
CTD
Dossier status
Under development
Country of origin
United States
GMP approvals
FDA
Comments
Indication: Friedreich's ataxia (FA) — individuals with confirmed GAA repeat expansion
Clinical stage: Phase 3 ongoing
Modality: Biologic (recombinant human frataxin fused to cell-penetrating peptide TAT)
Mechanism / Target: Delivers frataxin protein directly into cells — restores FXN in mitochondria, reducing iron accumulation and oxidative stress; addresses root cause of FA neurodegeneration
Route / form: Subcutaneous injection (daily)
Differentiation: Only frataxin protein replacement approach in Phase 3; addresses primary molecular defect; FDA Breakthrough Therapy designation; complementary to gene therapy approaches
Manufacturer #39185
Clinical-stage rare disease company focused on frataxin replacement therapy for Friedreich's ataxia. Develops cell-penetrating peptide-frataxin fusion protein to restore mitochondrial function in FA. Wholly owned programme with breakthrough therapy designation in this high-unmet-need disease.