Blincyto - Blinatumomab

What is Blincyto - Blinatumomab and what is it used for?

Blincyto is a cancer medicine indicated for the treatment of adults with acute lymphoblastic leukemia (ALL) from B-cell precursors, a type of blood cancer. In LLA from B cell precursors, some cells that give rise to B cells (a type of white blood cell) multiply too quickly to replace normal blood cells.

Blincyto is used when the ALL has returned or has not responded to previous treatments. It is used in patients with a "Philadelphia negative chromosome" (Ph-). This means that some of their genes have not reorganized to form a special chromosome, called "Philadelphia chromosome", which is present in some patients with ALL.

Blincyto contains the active ingredient blinatumomab.

Because the number of patients with ALL is low, the disease is considered "rare" and Blincyto was designated an "orphan medicine" (a medicine used in rare diseases) on 24 July 2009.

How is Blincyto used - Blinatumomab?

The medicine can only be obtained with a prescription and treatment should be started by a doctor experienced in treating patients with blood cancer.

Blincyto is available as a powder for solution for infusion (drip into a vein). Blincyto is administered by continuous infusion during a 4-week treatment cycle, using a pump device. Patients must remain in the hospital for at least 9 days in the first cycle and for at least 2 days in the second cycle. Each treatment cycle is separated from the next by a 2-week interval without treatment. Patients who achieve complete remission after 2 treatment cycles may be subjected to a maximum of 3 additional Blincyto cycles, if the benefits outweigh the risks for the individual subject.

Before receiving Blincyto, patients can be treated with medicines to avoid reactions to the infusion or the onset of fever. Furthermore, chemotherapy injections should be practiced in patients in the spinal region to prevent the spread of leukemia cells in the nervous system.

For more information, see the package leaflet.

How does Blincyto - Blinatumomab work?

The active substance in Blincyto, blinatumomab, is a type of antibody that was designed to recognize and bind to two proteins:

the CD19 protein, which is found on the surface of all B cells, including the ALL cells;
the CD3 protein, which is found on the surface of T cells (the cells of the immune system responsible for the destruction of pathogens and cancer cells).

Blincyto acts as a "bridge" to create a link between the T cells and the B cells. The T cells are therefore activated so that they release substances capable of destroying the B cells.

What benefit has Blincyto - Blinatumomab shown during the studies?

Blincyto was investigated in a main study involving 189 patients with negative B-cell precursor ALL for the Philadelphia chromosome, in whom the leukemia had recurred or failed to respond to treatment. Patients were treated with Blincyto up to a maximum of five treatment cycles. In this study, Blincyto was not compared to other therapies. The main measure of effectiveness was the percentage of patients who responded to treatment after two treatment cycles, showing a resolution of the signs of leukemia and a complete or partial normalization of blood counts. The study found that 42.9% (81 of 189) of patients treated with Blincyto responded to treatment. In most patients where a response has been observed, no residual tumor cells have been found. The average survival before a tumor reappeared was about 6 months, which allowed eligible patients to undergo a hematopoietic stem cell transplant.

What is the risk associated with Blincyto - Blinatumomab?

The most common side effects with Blincyto (which may affect more than 1 in 10 people) are infusion-related reactions (including fever, chills and tremors), infections, pyrexia (fever), headache, febrile neutropenia (decrease in the number of white blood cells accompanied by fever), peripheral edema (swelling, especially of the ankles and feet), nausea, hypokalaemia (decreased potassium levels in the blood), constipation, anemia (reduction in the number of red blood cells), cough, diarrhea, tremor, neutropenia, abdominal pain, insomnia, fatigue and chills.

The most serious side effects were infections, neurological events (including confusion, tremors, dizziness, numbness or tingling), neutropenia with or without fever, cytokine release syndrome (a complication due to the massive release of proinflammatory proteins in the blood) and tumor lysis syndrome (a complication due to the destruction of tumor cells). For the full list of all side effects reported with Blincyto, see the package leaflet.

The use of the medicine is contraindicated in breast-feeding women. For the full list of limitations, see the package leaflet.

Why has Blincyto - Blinatumomab been approved?

The Agency's Committee for Medicinal Products for Human Use (CHMP) decided that Blincyto's benefits are greater than its risks and recommended that it be approved for use in the EU. The committee noted that Blincyto is of benefit for the treatment of negative B-cell precursor ALL for the Philadelphia chromosome in high-risk adults who have few therapeutic options and who generally have a poor prognosis. However, since the main study did not compare Blincyto with other standard therapies, the CHMP considered it necessary to collect further data. The safety profile of the medicine was considered acceptable, provided the recommendations given are observed.

Blincyto has obtained a "conditional approval". This means that further information on the medicine, which the company is required to provide, will be available in the future. Every year the European Medicines Agency will review the new information available and this summary will be updated accordingly.

What information is still awaited for Blincyto - Blinatumomab?

Because a conditional approval has been issued for Blincyto, the company that markets Blincyto will provide data from a larger study comparing the efficacy of Blincyto with standard chemotherapy (anticancer medicines) in patients with precursor ALL. of B cells negative for the Philadelphia chromosome. An additional study will examine the safety and use of Blincyto in clinical practice.

What measures are being taken to ensure the safe and effective use of Blincyto - Blinatumomab?

A risk management plan has been developed to ensure that Blincyto 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 Blincyto, including the appropriate precautions to be followed by healthcare professionals and patients.

The company that markets Blincyto will also provide patients and healthcare professionals with informative material containing instructions on how to administer Blincyto and how to manage the risks of the medicine. Patients will also be given an alert card.