by Health Editor
Reimbursed in Italy for a drug with an innovative mechanism of action, used in the third line in patients who develop resistance or intolerance to previous therapies
The complexity of being forced, at a certain point in therapy, to change direction: this is the experience of those who live with chronic myeloid leukemia, a blood cancer that affects almost 9,000 Italians. In fact, despite therapeutic progress, a high number of patients have to change treatment because they develop resistance or intolerance to the treatment. To meet the needs of these patients, a new therapeutic option is now available, also reimbursed in Italy.
New treatment in the third line
The Italian drug agency (Aifa) has in fact recently approved asciminib, the progenitor of a new generation of drugs, the so-called stamp inhibitors (specifically targeting the Abl myristoyl pocket) which, thanks to its particular mechanism of action, is effective and well tolerated, with a positive impact on the quality of life of patients. The indication concerns patients with chronic myeloid leukemia with positive Philadelphia chromosome in the chronic phase, with resistance or intolerance to at least two previous tyrosine kinase inhibitors, drugs used for standard treatment. “Even today, many patients in the second line develop resistance or intolerance to therapy, which is why it is important to make available new therapeutic options in the third line that are effective, well tolerated and capable of guaranteeing a good quality of life – comments Fabrizio Pane, full professor at the Federico II University of Naples and director of Hematology and bone marrow transplants at the Federico II University Hospital of Naples —. The patients involved in an international survey in 11 countries, including Italy, indicate the importance of curbing the progression of the disease and achieving a good quality of life as the main objectives of treatment”.
Different mechanism of action
This blood cancer strikes mainly after the age of 60 and today, thanks to the development and use of tyrosine kinase inhibitors, it allows ten-year survivals of over 80%. This type of treatment is ongoing and in most cases is given for life to keep the cancer at bay. The great effectiveness of these treatments has almost eliminated the need for bone marrow transplantation, once the only treatment capable of curing patients. However, asciminib has a novel mechanism of action and is associated with fewer adverse events, with positive consequences for patients. “Unlike other tyrosine kinase inhibitors, the new drug binds highly specifically to the BCR-ABL1 tyrosine kinase, the switch that turns on the disease. Therefore, it is an effective treatment with a good tolerability profile: this is a very important aspect, given the risks to which patients may be exposed due to the long years of treatment and the frequent presence of other comorbidities (i.e. concomitant pathologies) » explains Fausto Castagnetti, associate professor at the University of Bologna and working at the Seràgnoli Institute of Hematology.
Safe, well tolerated, effective for a long time
«The efficacy of asciminib is confirmed by data from the pivotal phase three study ASCEMBL, which showed (after 24 weeks of treatment) a major molecular response rate, almost double (25.5% versus 13.2%), compared to another tyrosine kinase inhibitor, efficacy also confirmed at 96 weeks with a higher molecular response rate of 37.6% for asciminib against 15.8% for the other medicine» adds Castagnetti. Positive results are also confirmed by experience in clinical practice. “The phase one study evaluated, for a median duration of 4 years, the effects of asciminib in heavily pre-treated patients and demonstrated its long-term safety, tolerability and efficacy,” concludes Massimo Breccia, associate professor at the University La Sapienza of Rome and hematologist at the Policlinico Umberto I —. The Italian clinical experience, during the compassionate use program conducted between 2019 and 2023, then highlighted the efficacy of the drug in maintaining and improving the molecular response and its good tolerability profile. Furthermore, a survey by the GIMEMA Foundation investigated the perception of Italian haematologists on the use of this new therapeutic option: 89% would use it as a third-line treatment in resistant patients and 98% consider it, precisely because of its tolerability, a drug also for the elderly”.
August 11, 2023 (change August 11, 2023 | 07:26)
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