Trepulmix

Trepulmix is given by infusion (drip) under the skin using a pump to control the speed of infusion. The dose is calculated on the basis of the patient's weight and is adjusted according to how well the condition is controlled and the severity of any side effects. The patient will be trained on using the pump and on infusing the medicine.

The medicine can only be obtained with a prescription and treatment with Trepulmix should be started and monitored only by healthcare professionals experienced in treating pulmonary hypertension. Treatment should be started in a setting where intensive care facility is available.

For more information about using Trepulmix, see the package leaflet or contact your doctor or pharmacist.

Treprostinil, the active substance in Trepulmix, works in a similar way to prostacyclin, a natural substance that widens blood vessels and stops platelets (blood components) from sticking to each other to form blood clots. In patients with CTEPH, these effects of treprostinil prevent blood clots and lower blood pressure in the pulmonary artery and so improve symptoms of the disease.

A main study involving 105 patients with severe CTEPH who could not have an operation found that Trepulmix can improve patients’ capacity for physical activity, measured as the ability to walk.

In this study, patients received Trepulmix at a high dose (30 nanograms/kg/minute) or low dose (3 nanograms/kg/minute) which was not expected to have an effect. After 24 weeks, patients receiving the high dose were able to walk an average of 45 metres further in 6 minutes than at the start of treatment, compared with 4 metres further for those receiving the low dose.

Additional studies including comparison with records of patients with CTEPH who had not been treated with Trepulmix confirmed improvement in physical capacity.

The most common side effects with Trepulmix (which may affect more than 1 in 10 people) are headache, widening of blood vessels (with flushing), diarrhoea, nausea (feeling sick), jaw pain, reactions around the injection site such as pain, swelling and bleeding.

Trepulmix must not be used in patients with conditions like heart problems, stroke, problems in the gut such as ulcers, blocked veins in lungs and severe liver problems. Patients must not take Trepulmix at the same time as similar medicines called prostanoids.

For the full list of side effects and restrictions of Trepulmix, see the package leaflet.

The main study found that Trepulmix increases the distance patients can walk in 6 minutes. Although this study was small (because of the rarity of the disease), the results were meaningful. Additional studies showed that Trepulmix improved blood circulation and patients’ physical capability.

Side effects are those expected from the way Trepulmix works and are manageable. The European Medicines Agency therefore decided that Trepulmix’s benefits are greater than its risks and it can be authorised for use in the EU.

Recommendations and precautions to be followed by healthcare professionals and patients for the safe and effective use of Trepulmix have been included in the summary of product characteristics and the package leaflet.

As for all medicines, data on the use of Trepulmix are continuously monitored. Side effects reported with Trepulmix are carefully evaluated and any necessary action taken to protect patients.

Trepulmix received a marketing authorisation valid throughout the EU on 3 April 2020.