What is and what is Besponsa - Inotuzumab ozogamicin used for?
Besponsa is a cancer medicine used to treat a type of blood cancer that affects B cells (a type of white blood cell) called acute lymphoblastic leukemia (ALL) from cell precursors B. Besponsa is used on its own in adults whose cancer has resumed or failed to respond to a previous treatment.
Besponsa is used exclusively in patients with "CD22-positive B-cell precursor ALL". This means that patients have a specific protein (CD22) on the surface of white blood cells. In patients who have a type of chromosome known as the Philadelphia chromosome, treatment with an anticancer medicine called tyrosine kinase inhibitor should be tried before administering Besponsa.
Because the number of patients with B-cell precursor ALL is low, the disease is considered 'rare' and Besponsa was designated an 'orphan medicine' (a medicine used in rare diseases) on 7 June 2013.
esponsa contains the active substance inotuzumab ozogamicin
How is Besponsa - Inotuzumab ozogamicin used?
Besponsa is given by infusion (drop by drop) into a vein over a period of at least an hour. Infusions are given on days 1, 8 and 15 of a 3 or 4 week treatment cycle. Your doctor may stop treatment or reduce your dose if the patient experiences certain serious side effects.
Patients in whom Besponsa works well must receive 2 or 3 cycles, after which they can undergo a stem cell transplant to replace bone marrow, the only curative treatment. Patients whose treatment works well but who will not undergo a stem cell transplant can receive up to a maximum of 6 treatment cycles. In patients who do not respond to treatment, Besponsa should be discontinued after 3 cycles.
Besponsa can only be obtained with a prescription and treatment must be administered under the supervision of a doctor experienced in the use of anticancer treatments.
For more information, see the package leaflet.
How does Besponsa - Inotuzumab ozogamicin work?
The active substance in Besponsa, inotuzumab ozogamicin, is a monoclonal antibody (a type of protein) that has been linked to a small molecule, N-acetyl-gamma-calyceamicin dimethylhydrazide. The monoclonal antibody was designed to recognize and bind to CD22 on tumor B cells. Once bound, the medicine is absorbed by the cell where the caliceamicina becomes active, causing breakage in the cellular DNA and thus killing the cancer cell.
What benefit has Besponsa - Inotuzumab ozogamicin shown during the studies?
Besponsa has been shown to be more effective than other chemotherapy (medicines to treat tumors) in a main study involving 326 adults with CD22-positive B-cell precursor ALL, who had recurred or had not responded to a previous treatment. The main indicator of effectiveness was the response to treatment.
Patients were considered to have responded if they did not present with residual tumor B cells in the blood and bone marrow after treatment. An analysis of the first 218 treated patients showed that, after at least 2 treatment cycles, 81% (88 out of 109) of patients receiving Besponsa responded to treatment compared to 29% (32 of 109) of patients receiving other chemotherapy. Patients who responded to the treatment could be subjected to a stem cell transplant.
What are the risks associated with Besponsa - Inotuzumab ozogamicin?
The most common side effects with Besponsa (which may affect more than 1 in 5 people) are thrombocytopenia (low platelet count in the blood), neutropenia and leukopenia (low white blood cell count), infection, anemia (low red blood cell count), tiredness, hemorrhage (bleeding), fever, nausea (feeling sick), headache, febrile neutropenia (low white blood cell count associated with fever), abdominal pain (stomach ache), increased levels of liver enzymes called transaminases and gamma -glutamyltransferase, and hyperbilirubinemia (elevated blood levels of bilirubin, a breakdown product of red blood cells).
The most serious side effects are infection, febrile neutropenia, haemorrhage, abdominal pain, fever, fatigue and veno-occlusive liver disease / sinusoidal obstruction syndrome (VOD / SOS, a serious liver disease).
Besponsa should not be used in patients who have VOD / SOS or have had severe VOD / SOS or have other severe liver diseases.
For the full list of restrictions and side effects reported with Besponsa, see the package leaflet.
Why has Besponsa - Inotuzumab ozogamicin been approved?
The Agency's Committee for Medicinal Products for Human Use (CHMP) decided that Besponsa's benefits are greater than its risks and recommended that it be approved for use in the EU.
The CHMP considered that, although there has recently been progress in the treatment of B-cell precursor ALL, treatment options for patients remain limited. The main study showed that Besponsa was better than other commonly used chemotherapy drugs because it induced a response in patients and allowed them to undergo a curative stem cell transplant.
Regarding safety, the side effects of Besponsa are similar to those of other chemotherapy drugs and can generally be managed with a dose reduction or treatment interruption.
What measures are being taken to ensure the safe and effective use of Besponsa - Inotuzumab ozogamicin?
Recommendations and precautions to be followed by healthcare professionals and patients for the safe and effective use of Besponsa have been included in the summary of product characteristics and the package leaflet.
Other information on Besponsa - Inotuzumab ozogamicin
The full EPAR for Besponsa can be found on the Agency's website: ema.europa.eu/Find medicine / Human medicines / European public assessment reports. For more information about Besponsa therapy, read the package leaflet (also part of the EPAR) or contact your doctor or pharmacist.
The summary of the opinion of the Besponsa Committee for Orphan Medicinal Products is available on the Agency's website: ema.europa.eu/Find medicine / Human medicines / Rare disease designation.
