pp-150 Solution for injection
Dossier type
CTD
Dossier status
Ready to file
Country of origin
United States
GMP approvals
FDA
Comments
Indication: Hereditary angioedema (HAE) — long-term prophylaxis
Clinical stage: FDA approved 2025; post-marketing studies ongoing
Modality: Oligonucleotide (GalNAc-conjugated antisense, ASO)
Mechanism / Target: Silences hepatic KLKB1 mRNA (plasma kallikrein precursor) — reduces plasma kallikrein activity and bradykinin production
Route / form: Subcutaneous injection (monthly)
Differentiation: Monthly self-injection convenience; KLKB1 silencing complements lanadelumab (plasma kallikrein protein inhibitor) and NTLA-2002 (KLKB1 gene knockout) in same pathway
Manufacturer #39180
Leader in RNA-targeting therapeutics with multiple approved antisense drugs. Develops GalNAc-conjugated ASOs for precise liver-targeted gene silencing in cardiometabolic and rare diseases. Wholly owned commercial and late-stage pipeline with independent global development.
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