MHRA Approves Plozasiran (Redemplo) for Adults Battling Familial Chylomicronaemia Syndrome

The Medicines and Healthcare products Regulatory Agency (MHRA) has granted approval for plozasiran (Redemplo) on 19 October 2026, aimed at lowering triglyceride levels in adults diagnosed with familial chylomicronaemia syndrome (FCS), in conjunction with a low-fat diet.

Understanding Familial Chylomicronaemia Syndrome

Familial chylomicronaemia syndrome is a rare genetic disorder that leads to dangerously high levels of triglycerides in the bloodstream. Such elevated levels can result in inflammation of the pancreas, a condition known as pancreatitis, which is characterised by severe abdominal pain and other complications. Managing this condition is crucial to prevent potentially life-threatening outcomes.

Plozasiran functions by inhibiting the production of apolipoprotein C3, a liver-produced protein that impedes the breakdown of fats. By reducing the levels of this protein, plozasiran effectively lowers triglyceride concentrations in the blood.

Administration and Treatment Protocol

The medication is administered via a 25 mg injection beneath the skin, to be given once every three months. Patients or their caregivers are trained to self-administer the injection following proper guidance, ensuring a convenient treatment process.

Julian Beach, Executive Director of Operations, remarked on the significance of this authorisation, highlighting that it offers an additional therapeutic option for adults suffering from this rare and painful condition. He noted, “Clinical trial data demonstrated that plozasiran significantly reduced triglyceride levels when used alongside a controlled diet. As with all medications, we will maintain vigilant monitoring of the safety profile of plozasiran.”

Clinical Evidence and Study Outcomes

The authorisation was supported by a principal study involving 75 adults with FCS, all of whom adhered to a controlled diet. Of these participants, 50 received plozasiran, while 25 were administered a placebo. Over a 10-month period, those treated with plozasiran experienced a median reduction in triglyceride levels of approximately 80%, compared to just 17% in the placebo group. Additionally, the study recorded a decrease in the incidence of acute pancreatitis among participants using plozasiran.

Side Effects and Reporting Procedures

The most frequently reported side effect associated with plozasiran is hyperglycaemia, affecting more than one in ten users. Other common side effects, which may impact up to ten per cent of individuals, include headaches, nausea, and local reactions at the injection site. It is important for anyone experiencing adverse effects to consult their healthcare provider and report their experiences through the Yellow Card scheme available at https://yellowcard.mhra.gov.uk or via the MHRA Yellow Card app.

Regulatory Context and Further Information

The marketing authorisation for plozasiran was granted to Arrowhead Pharmaceuticals Ireland Limited on 19 October 2026. This approval was obtained through the International Recognition Procedure (IRP), Route B, with the European Medicines Agency (EMA) serving as the reference regulatory authority.

Further details regarding plozasiran can be found in the Summary of Product Characteristics (SmPC) and Patient Information Leaflet (PIL), which will be accessible on the MHRA Products website in due course.

The MHRA plays a vital role in the regulation of all medicines and medical devices within the UK, ensuring they are effective and safe for use. The agency’s work is grounded in thorough and evidence-based assessments to guarantee that the benefits of any treatment outweigh the associated risks.

As an executive agency of the Department of Health and Social Care, the MHRA remains committed to protecting public health through stringent regulatory practices.

For media inquiries, please reach out to newscentre@mhra.gov.uk or call 020 3080 7651.