Unituxin - Dinutuximab

What is Unituxin - Dinutuximab used for?

Unituxin is a cancer medicine used to treat neuroblastoma, a cancer of nerve cells, in children aged 12 months to 17 years.

Unituxin is used to treat children with "high-risk" neuroblastoma, the form of cancer that has a high probability of recurrence. Children treated with Unituxin must first have responded to chemotherapy and then received additional treatment for bone marrow cleansing (myeloablative therapy) and a stem cell transplant.

Unituxin is used in combination with 3 other medicines: GM-CSF, interleukin-2 and isotretinoin.

Because the number of patients with neuroblastoma is low, the disease is considered "rare" and Unituxin was designated an "orphan medicine" (a medicine used for rare diseases) on 21 June 2011.

Unituxin contains the active ingredient dinutuximab.

How is Unituxin - Dinutuximab used?

Unituxin is given by infusion (drip) into a vein. The daily dose depends on the child's body surface area and infusions are administered over 10 hours. The patient also receives 3 other medicines: isotretinoin, GM-CSF and interleukin-2. The treatment lasts about 6 months, but not all medicines are given every month. Unituxin is given for four consecutive days every month, in the first 5 months.

Due to the risk of severe allergic reactions with Unituxin, equipment and personnel must be immediately available for resuscitation of the patient in case such reactions occur. Patients should also be given an antihistamine before starting each Unituxin infusion, in order to reduce the risk of reactions.

Because pain is a common side effect of treatment with Unituxin, patients are also given painkillers.

Unituxin is for hospital use only and treatment should be performed under the supervision of a doctor experienced in cancer therapies. The medicine can only be obtained with a prescription

How does Unituxin - Dinutuximab work?

The active substance in Unituxin, dinutuximab, is a monoclonal antibody designed to recognize and bind to a substance present at high levels in neuroblastoma cancer cells, known as GD2 ganglioside. When dinutuximab binds to gangliosides on neuroblastoma cells, it marks cells as targets for the immune system (the body's natural defenses), which then attacks them. In this way, the medicine can help eliminate cancer cells left in the body after other treatments.

What benefit has Unituxin - Dinutuximab shown during the studies?

In a main study conducted in 230 patients with high-risk neuroblastoma, Unituxin (administered with isotretinoin, GM-CSF and interleukin-2) was more effective than isotretinoin alone for patient survival and for preventing recurrence of cancer. After about 3 years, 80% of patients treated with Unituxin were alive, compared with 67% of patients treated with isotretinoin alone.

What is the risk associated with Unituxin - Dinutuximab?

The most common side effects with Unituxin (seen in more than 30% of patients) are: pain affecting any part of the body, hypotension (low blood pressure), hypersensitivity (allergic reactions), fever, urticaria, syndrome of increased capillary permeability ( a disease characterized by the leakage of fluids from blood vessels, which causes swelling and drop in blood pressure), anemia (low number of red blood cells in the blood), low number of platelets, low levels of sodium and potassium, increase in liver enzymes and low levels of white blood cells. For the full list of side effects and limitations, see the package leaflet.

Why has Unituxin - Dinutuximab been approved?

Patients with high-risk neuroblastoma need aggressive therapy, which is often not sufficient to prevent tumor recurrence. A study conducted with Unituxin in combination with isotretinoin, GM-CSF and interleukin-2 showed that the association can improve outcomes in these patients, prolonging their survival and helping to prevent recurrence or worsening of the disease.

Although undesirable effects with Unituxin may be severe and medications are needed to prevent allergic reactions and pain, the risks of the medicine are considered acceptable given the severity of the disease. The number of patients who have stopped treatment due to side effects appears to be low and these effects can be managed with appropriate measures.

The Agency's Committee for Medicinal Products for Human Use (CHMP) decided that Unituxin's benefits are greater than its risks and recommended that it be approved for use in the EU.

What measures are being taken to ensure the safe and effective use of Unituxin - Dinutuximab?

A risk management plan has been developed to ensure that Unituxin is used as safely as possible. Based on this plan, safety information has been included in the summary of product characteristics and the package leaflet for Unituxin, including the appropriate precautions to be followed by healthcare professionals and patients.

In addition, the company that markets Unituxin will conduct two studies to learn more about the safety of the medicine, even in the long term.