Earlier, ARO-APOC3 received Orphan Drug designation from the FDA and the European Union.
ARO-APOC3 is the firm’s investigational RNAi therapeutic targeting apolipoprotein C-III (APOC3).
It is being developed as a therapy for patients suffering with severe hypertriglyceridemia (SHTG), mixed dyslipidemia (MD), and FCS.
A rare genetic disorder, FCS causes severely high triglyceride levels, which can lead to acute and potentially fatal pancreatitis.
Currently, there are no therapies approved by the FDA to treat FCS.
The Phase 3 PALISADE clinical study (NCT05089084) is investigating ARO-APOC3 in patients with FCS.
ARO-APOC3 is also being studied in the Phase 2 SHASTA-2 clinical study (NCT04720534) in patients with SHTG, as well as in the Phase 2 MUIR clinical study (NCT04998201) in patients with MD.
Arrowhead Pharmaceuticals develops medicines to treat intractable diseases by gene silencing approach.
Leveraging a wide portfolio of RNA chemistries and effective modes of delivery, Arrowhead’s treatment triggers the RNA interference system to lead to the quick and durable knockdown of target genes.