Eurpean Commission grants marketing authorisation for Redemplo (plozasiran) to reduce triglycerides in adults with familial chylomicronemia syndrome – Arrowhead
Arrowhead Pharmaceuticals, Inc. announced that the European Commission (EC) has formally granted marketing authorization for Redemplo (plozasiran), a small interfering RNA (siRNA) medicine, as an adjunct to diet to reduce triglyceride levels in adult patients with familial chylomicronemia syndrome (FCS). Redemplo is the first and only siRNA medicine authorized by the EC for adults with FCS, diagnosed either by the presence of clinical criteria or genetic testing.
“FCS drives an elevated risk of recurrent and potentially fatal episodes of acute pancreatitis. Results from the PALISADE study demonstrate that plozasiran can achieve significant and sustained reductions in triglycerides for these patients,” said Professor Børge Nordestgaard, Department of Clinical Medicine, University of Copenhagen and President, European Atherosclerosis Society. “Importantly, the ability to diagnose and treat without requiring a genetic test could enable earlier treatment, which is particularly relevant in rare diseases such as FCS.”
Announcing its recommendation for the approval of Redemplo, the European Medicines Agency noted, “Although other authorised medicines can help people with FCS confirmed by genetic testing, Redemplo does not require genetic confirmation of the condition, thus providing a treatment option for more adults with FCS and addressing the unmet medical need in these patients.”
“Today’s approval marks a pivotal moment for people living with familial chylomicronemia syndrome. As a patient myself and having spoken with countless others living with FCS through leading our organization, I know firsthand how devastating the burden of FCS is on every dimension of daily life. The constant uncertainty, the worry, the fear of acute pancreatitis, the chronic pain and fatigue are challenges the FCS community faces every single day, on top of the long and often frustrating journey to receiving a diagnosis,” added Rosa Pérez Jiménez, President of Familial Chylomicronemia Association (Asociación de Quilomicronemia Familiar) Spain. “This new therapeutic option gives renewed hope to patients who have waited far too long to be seen, understood, and treated.”
Harnessing Arrowhead’s proprietary Targeted RNAi Molecule (TRiM) platform, Redemplo is designed to suppress production of apolipoprotein C-III (APOC3), a protein produced in the liver that raises triglyceride levels by inhibiting their breakdown and clearance.
“We are pleased to have received EC approval for Redemplo as a new treatment option for people living with genetically or clinically confirmed FCS. With this approval secured, we are engaging with relevant national authorities and healthcare communities across the European Union to bring Redemplo to people living with FCS as quickly and efficiently as possible,” said Christopher Anzalone, Ph.D., President and CEO at Arrowhead Pharmaceuticals. “This ongoing cadence of regulatory approvals around the world reflects the strength of our clinical data and the real progress being made across our diverse pipeline of siRNA-based therapies that leverage our proprietary TRiM platform.”
EC regulatory approval was supported by clinical data from the Phase III PALISADE study, a randomized, double-blind, placebo-controlled trial in 75 adults with clinically diagnosed or genetically confirmed FCS. The PALISADE study met its primary endpoint and all multiplicity-controlled key secondary endpoints. In PALISADE, 25 mg Redemplo reduced triglycerides by a median of 80% from baseline versus a 17% reduction with placebo. Additionally, the combined doses of 25 mg and 50 mg plozasiran significantly reduced the incidence of acute pancreatitis (odds ratio, 0.169; p=0.0292). The odds of acute pancreatitis were 83% lower in the pooled plozasiran groups compared with the placebo group. The most common adverse reactions were hyperglycaemia (12.8%), headache (6.8%), nausea (4.7%), and injection site reaction (4.7%).
