The Medicines and Healthcare products Regulatory Agency (MHRA) has authorised plozasiran (Redemplo), an injection marketed by Arrowhead Pharmaceuticals Ireland, for adults with familial chylomicronaemia syndrome (FCS), a rare condition that causes abnormally high levels of triglycerides in the blood.
FCS can lead to inflammation of the pancreas, known as pancreatitis, causing severe abdominal pain.
Plozasiran blocks the production of apolipoprotein C3, a protein made in the liver that slows the breakdown of fats. This help in lowering triglyceride levels in the blood.
The medicine is administered as a 25 mg injection under the skin once every three months. Patients or their caregivers can give the injection after receiving training.
Plozasiran was approved through the International Recognition Procedure (IRP), Route B, with the European Medicines Agency (EMA) as the reference regulator.
Julian Beach, Executive Director, Operations, said: “This authorisation provides an additional treatment option for adults with familial chylomicronaemia syndrome, a rare condition that can cause serious and painful complications.
“Clinical trial data showed that plozasiran substantially reduced triglyceride levels when used alongside a controlled diet.
“As with all medicines, we will continue to monitor the safety of plozasiran closely.”
The authorisation is supported by a main study involving 75 adults with FCS. All participants followed a controlled diet, with 50 receiving plozasiran and 25 receiving placebo.
After 10 months, participants receiving plozasiran had a median reduction in triglyceride levels of around 80%, compared with 17% among those receiving placebo.
The study also recorded fewer episodes of acute pancreatitis in participants receiving plozasiran.
The most common side effect reported with plozasiran is hyperglycaemia (raised blood sugar), which may affect more than one in 10 people.
Other common side effects include headache, nausea and reactions at the injection site.
