The Italian Medicines Agency has approved the reimbursement for the monotherapy treatment of adult patients with newly diagnosed acute myeloid leukemia who are not eligible for standard induction chemotherapy. This is a fixed-dose oral therapy of decitabine 35 mg and cedazuridine 100 mg. The Italian regulatory body confirms the EMA’s decision from last year.
“Acute myeloid leukemia is a rare malignant tumor that affects hematopoietic stem cells,” says Prof. Elizabeth TodiscoDirector of the Hematology and Stem Cell Transplant Department of the Busto Arsizio Hospital, ASST Valle Olona. It is estimated that there are over 3,200 new cases per year in our country and the average age of onset is 68 years. The disease progresses rapidly and is extremely disabling. Therefore, it has a strong impact on the quality of life of the individual patient and determines a significant use of social and health resources. The new therapy could guarantee important benefits because the patient is no longer forced to go to treatment centers to receive the treatment. The drug is oral and the method of administration also favors therapeutic adherence. Furthermore, access to health facilities is reduced and therefore also the complicated management of hospital infections, which are very frequent among immunosuppressed people”.
The new medicinal product based on decitabine and cedazuridine was evaluated in a randomized, crossover phase 3 clinical study involving 89 patients with AML who were not eligible for standard induction chemotherapy. Participants were randomized 1:1 to receive oral decitabine/cedazuridine in cycle 1 and intravenous decitabine in cycle 2 or the reverse sequence. From the third cycle onwards, all patients, regardless of the previous sequence, were treated with the oral decitabine/cedazuridine combination. The study showed a pharmacokinetic and pharmacodynamic profile similar to intravenous decitabine. Pharmacokinetic equivalence was 99.64%. Median overall survival was 8.9 months while the complete response rate was 21.8%.
“No significant differences emerged in the safety profile of oral decitabine/cedazuridine vs. IV decitabine,” continues Professor Todisco. “This increases the therapeutic opportunities for patients and clinicians in our country against an extremely complex hematological disease. In fact, we are seeing constant improvements in the treatment of the disease, but overall 5-year survival is only 15%. The combination of decitabine and cedazuridine guarantees a good quality of life, thanks above all to its oral formulation and greater independence from transfusion therapies.”
“Since 2009, our company has been operating in Italy with the utmost commitment to making available new drugs capable of combating serious diseases, such as acute myeloid leukemia,” concludes Dr. Alessandro LattuadaCEO of Otsuka Pharmaceutical Italy -. We are pleased with the recent decision by AIFA that allows Italian patients access to this new therapeutic option that promises to change the current management of the disease, while improving the quality of life of the patient and the caregiver”.
