FDA Approves Oral Combination Therapy for Older Adults With AML

The Food and Drug Administration (FDA) has announced the approval of an all-oral combination therapy for adults newly diagnosed with acute myeloid leukemia (AML), offering a new treatment pathway for patients who are unable to undergo intensive chemotherapy due to age or other health factors.

The approval, granted on May 13, covers the use of Inqovi (decitabine and cedazuridine) tablets in combination with Venclexta (venetoclax). This regimen is specifically indicated for adults aged 75 years or older, or those with comorbidities that preclude the use of standard, intensive induction chemotherapy.

For patients facing an AML diagnosis later in life, the availability of an oral regimen represents a significant shift in the treatment landscape. Standard treatment for AML often involves high-intensity chemotherapy that requires lengthy hospital stays and carries a high risk of severe side effects, which many older or medically frail patients cannot tolerate.

Clinical trial results

The FDA based its decision on results from Study ASTX727-07, a single-arm, open-label clinical trial. The study evaluated 101 adult patients with newly diagnosed AML who met the criteria for being unable to receive intensive chemotherapy.

Data from the trial showed that 41.6% of participants (42 out of 101 patients) achieved complete remission (CR). The median time for patients to reach complete remission was two months, with a range spanning from 0.4 to 15.3 months.

Researchers also measured the duration of complete remission (DoCR), defined as the period from the first instance of CR until the disease relapsed or the patient died. Notably, the median duration of complete remission was not reached during the study period, though observed durations ranged from 0.5 to 16.3 months.

Dosage and administration

The newly approved regimen simplifies administration by utilizing a tablet form of decitabine and cedazuridine. The recommended dosage for Inqovi, when used alongside Venclexta, is one tablet (containing 35 mg of decitabine and 100 mg of cedazuridine) taken once daily.

Patients are directed to take the tablet on Days 1 through 5 of each 28-day cycle. This treatment schedule is intended to continue until the disease progresses or the patient experiences unacceptable toxicity levels.

Safety and side effects

While the oral combination provides a less intensive alternative to traditional chemotherapy, the FDA included several warnings and precautions in the drug’s prescribing information.

The primary safety concerns include myelosuppression, a condition where bone marrow activity is decreased, resulting in fewer red blood cells, white blood cells and platelets. Additionally, the treatment carries a warning regarding embryo-fetal toxicity, indicating that the drugs could cause harm to a developing fetus.

Healthcare professionals and patients are encouraged to report any serious adverse events to the FDA’s MedWatch Reporting System.

Global collaboration

The review of this drug combination was conducted under Project Orbis, an initiative led by the FDA Oncology Center of Excellence. Project Orbis provides a framework for the concurrent submission and review of oncology products among international regulatory partners. For this specific approval, the FDA collaborated with Health Canada. While the treatment is now approved in the United States, the FDA noted that applications may still be under review by other international regulatory agencies.

Full prescribing information for the Inqovi and venetoclax combination will be made available through the Drugs@FDA database.

Editor's note: This article is for informational purposes only and is not a substitute for professional medical advice, as your own experience will be unique. Use this article to guide discussions with your oncologist. Content was generated with AI, reviewed by a human editor, but not independently verified by a medical professional.

Reference

“FDA approves oral combination of decitabine and cedazuridine tablets with venetoclax for newly diagnosed acute myeloid leukemia,” news release.

For more news on cancer updates, research and education, don’t forget to subscribe to CURE®’s newsletters here.