Adakveo

Adakveo is given by infusion (drip) into a vein lasting 30 minutes and the dose depends on the patient’s weight. The first two infusions are given 2 weeks apart. Subsequent infusions are given every 4 weeks.

The medicine can only be obtained with a prescription. Treatment should be started by a doctor experienced in treating sickle cell disease.

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

The active substance in Adakveo, crizanlizumab, is a monoclonal antibody (a type of protein) designed to attach to a substance, P-selectin, present on the surface of the cells lining blood vessels. P-selectin helps cells stick to the blood vessels and plays a role in the clogging up of vessels during painful crises in sickle cell disease. By attaching to and blocking the action of P-selectin, the medicine helps prevent painful crises.

A main study in 198 patients with sickle cell disease showed that Adakveo was effective at reducing the number of painful crises. In this study, patients given Adakveo had on average 1.6 crises per year, while patients given placebo (a dummy treatment) had on average 3 crises per year.

The study also showed that Adakveo reduced the yearly number of crises by almost a third in patients already taking hydroxycarbamide (2.4 versus 3.6) and by half in patients not taking hydroxycarbamide(1 versus 2).

The most common side effects with Adakveo (which may affect more than 1 in 10 people) are joint pain, nausea, back pain, fever and abdominal (belly) pain. Severe joint pain or fever may occur in around 1 in 100 people.

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

The main study showed that Adakveo was effective at reducing the number of painful crises in patients with sickle cell disease. There was some uncertainty about the size of Adakveo’s effect because of how the study was designed, but results showed consistent improvements with Adakveo, including a reduction in hospitalisations.

The side effects of Adakveo were relatively mild and considered manageable. The European Medicines Agency concluded that Adakveo’s benefits are greater than its risks and it can be authorised for use in the EU.

Adakveo has been given ‘conditional authorisation’. This means that there is more evidence to come about the medicine, which the company is required to provide. Every year, the Agency will review any new information that becomes available and this overview will be updated as necessary.

Since Adakveo has been given conditional authorisation, the company that markets Adakveo will provide further data on the safety and effectiveness of the medicine from two additional studies.

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

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

Adakveo received a conditional marketing authorisation valid throughout the EU on 28 October 2020.