Pepaxti is a medicine used to treat adults with multiple myeloma (a cancer of the bone marrow) when the cancer has not responded to previous treatments (refractory).
It is used in combination with dexamethasone (an anti-inflammatory medicine) in adults who have received at least three prior therapies, including an immunomodulatory agent, a proteasome inhibitor and an anti‑CD38 antibody, and whose disease has worsened since the last treatment.
For patients who have had an autologous stem cell transplantation (a procedure where the patient’s bone marrow is cleared of cells and replaced by stem cells from the patients themselves), Pepaxti can be used if the time from transplantation to when the cancer comes back is at least three years.
Multiple myeloma is rare, and Pepaxti was designated an ‘orphan medicine’ (a medicine used in rare diseases) on 19 March 2015. Further information on the orphan designation can be found here: https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-15-1463
Pepaxti contains the active substance melphalan flufenamide.
Pepaxti : EPAR - Medicine overview (PDF/136.95 KB)
First published: 22/08/2022
Pepaxti : EPAR - Risk management plan summary (PDF/150.48 KB)
First published: 22/08/2022
|Agency product number||
Melphalan flufenamide hydrochloride
|International non-proprietary name (INN) or common name||
|Therapeutic area (MeSH)||
|Anatomical therapeutic chemical (ATC) code||
|Date of issue of marketing authorisation valid throughout the European Union||
27/11/2023 Pepaxti - EMEA/H/C/005681 - IAIN/0007
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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).
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Pepaxti is indicated, in combination with dexamethasone, for the treatment of adult patients with multiple myeloma who have received at least three prior lines of therapies, whose disease is refractory to at least one proteasome inhibitor, one immunomodulatory agent, and one anti-CD38 monoclonal antibody, and who have demonstrated disease progression on or after the last therapy. For patients with a prior autologous stem cell transplantation, the time to progression should be at least 3 years from transplantation (see section 4.4).