Reblozyl
Authorised
This medicine is authorised for use in the European Union
luspatercept
MedicineHumanAuthorised
Overview
Reblozyl is a medicine used to treat anaemia (low red blood cell counts) in adults with the following blood disorders:
- Myelodysplastic syndromes, a group of conditions where too few blood cells are produced by the bone marrow. Reblozyl is used in patients who need regular blood transfusions and who have a very low to moderate risk of their condition developing into acute myeloid leukaemia (a blood cancer) or of dying. It is used in patients in whom another medicine, erythropoietin, is unsuitable or does not work.
- Beta thalassaemia, a genetic condition in which patients cannot make enough beta globin, a component of haemoglobin (the protein in red blood cells that carries oxygen around the body).
These diseases are rare, and Reblozyl was designated an ‘orphan medicine’ (a medicine used in rare diseases). Further information on the orphan designations can be found on the European Medicines Agency’s website (myelodysplastic syndromes: 22 August 2014; beta thalassaemia: 29 July 2014).
The medicine contains the active substance luspatercept.
The medicine is available for injection under the skin and can only be obtained with a prescription. Treatment should be started by a doctor experienced in treatment of blood diseases.
The injection is given under the skin of the upper arm, thigh or belly. The recommended dose depends on the patient’s weight and is adjusted depending on the patient’s response. Treatment is given once every 3 weeks. If the patient develops serious side effects, treatment should be delayed until side effects have improved.
For more information about using Reblozyl, see the package leaflet or contact your doctor or pharmacist.
The active substance in Reblozyl, luspatercept, regulates the maturation of red blood cells. It does this by blocking a signalling pathway called Smad2/3 that slows down the maturation of red blood cells and is overactive in patients with beta thalassaemia and myelodysplastic syndromes. Blocking Smad2/3 increases the production of red blood cells and allows them to develop normally.
Myelodysplastic syndromes
The main study involved 229 adults with myelodysplastic syndromes requiring regular blood transfusions. Patients received either Reblozyl or placebo (a dummy treatment) in addition to normal standard of care. 58 out of 153 patients (38%) taking Reblozyl did not need a blood transfusion for at least 8 weeks compared with 10 out of 76 (13%) patients receiving placebo.
Beta thalassaemia
The main study involved 336 patients with beta thalassaemia requiring regular blood transfusions. Patients received either Reblozyl or placebo in addition to standard treatment. Blood transfusion needs were reduced by at least one third (33%) in 48 out of 224 patients (21%) taking Reblozyl compared with 5 out of 112 (4.5%) patients receiving placebo.
A second main study involved patients with beta thalassaemia not requiring regular blood transfusions. Patients received either Reblozyl or placebo together with standard treatment for at least 48 weeks. After 12 weeks, 74 out of 96 patients receiving Reblozyl (77%) had a rise of at least 1 g/dL in their haemoglobin level over the next 12 weeks without needing transfusions, compared with none of the 49 patients who received placebo.
The most common side effects with Reblozyl in patients with myelodysplastic syndromes (which may affect more than 15 in 100 people) are tiredness, diarrhoea, weakness, nausea (feeling sick), dizziness, back pain and headache. The most common serious or severe side effects (affecting more than 2 in 100 people) include syncope (fainting), tiredness, hypertension (high blood pressure), weakness, urinary tract infection (infection of the structures that carry the urine) and back pain.
The most common side effects of Reblozyl in patients with beta thalassaemia requiring transfusion (which may affect more than 15 in 100 people) are headache, bone pain and joint pain. The most common serious or severe side effects are hyperuricaemia (high levels of uric acid in the blood), stroke, effects due to blood clots in the veins such as deep vein thrombosis, portal vein thrombosis (clots in the veins supplying the liver) and pulmonary embolism (clots in the veins supplying the lungs).
The most common side effects of Reblozyl in patients with beta thalassaemia not requiring regular transfusion (which may affect more than 15 in 100 people) are headache, bone, back and joint pain, and prehypertension and hypertension (high blood pressure). The most common serious or severe side effect is traumatic fracture (a fracture caused for example by a fall or an accident).
Reblozyl must not be given during pregnancy. Women who can become pregnant must use effective contraception during treatment and for at least 3 months after the last dose. Patients requiring treatment to control the growth of extramedullary haemopoiesis masses (the formation of blood cells outside the bone marrow) must not use Reblozyl. For the full list of side effects and restrictions of Reblozyl, see the package leaflet.
Treatment with frequent blood transfusions can lead to accumulation of iron in the body, which can damage organs. Reblozyl reduces the need for blood transfusions in myelodysplastic syndromes and beta thalassaemia while its side effects are considered manageable. In patients with beta thalassaemia not requiring regular transfusions, higher haemoglobin levels are expected to improve outcomes for patients. The European Medicines Agency therefore decided that Reblozyl’s benefits are greater than its risks and it can be authorised for use in the EU.
The company that markets Reblozyl will provide educational packs for doctors who prescribe Reblozyl explaining that the medicine can be harmful to the unborn child and detailing the steps that need to be taken for the medicine to be used safely. It will also supply cards to women who can become pregnant about the safety measures they should take to avoid pregnancy.
Recommendations and precautions to be followed by healthcare professionals and patients for the safe and effective use of Reblozyl have also been included in the summary of product characteristics and the package leaflet.
As for all medicines, data on the use of Reblozyl are continuously monitored. Side effects reported with Reblozyl are carefully evaluated and any necessary action taken to protect patients.
Reblozyl received a marketing authorisation valid throughout the EU on 25 June 2020.
Product information
Latest procedure affecting product information: II/0023
11/01/2024
This medicine’s product information is available in all official EU languages.
Select 'available languages' to access the language you need.
Product information documents contain:
- summary of product characteristics (annex I);
- manufacturing authorisation holder responsible for batch release (annex IIA);
- conditions of the marketing authorisation (annex IIB);
- labelling (annex IIIA);
- package leaflet (annex IIIB).
Product details
- Name of medicine
- Reblozyl
- Active substance
- Luspatercept
- International non-proprietary name (INN) or common name
- luspatercept
- Therapeutic area (MeSH)
- Anemia
- Myelodysplastic Syndromes
- beta-Thalassemia
- Anatomical therapeutic chemical (ATC) code
- B03XA06
Pharmacotherapeutic group
Other antianemic preparationsTherapeutic indication
Reblozyl is indicated for the treatment of adult patients with transfusion-dependent anaemia due to very low, low and intermediate-risk myelodysplastic syndromes (MDS) with ring sideroblasts, who had an unsatisfactory response to or are ineligible for erythropoietin-based therapy (see section 5.1).
Reblozyl is indicated in adults for the treatment of anaemia associated with transfusion dependent and non transfusion dependent beta thalassaemia (see section 5.1).
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