The FDA on Tuesday approved an oral formulation of decitabine and cedazuridine (Inqovi) for adults with intermediate- and high-risk myelodysplastic syndromes (MDS) and chronic myelomonocytic leukemia (CMML).

The oral tablet is indicated for de novo or secondary MDS in patients with intermediate- and high-risk disease, per the International Prognostic Scoring System, and with the following French-American-British subtypes: refractory anemia alone, with ringed sideroblasts or excess blasts, and CMML.

"The FDA remains committed to providing additional treatments to patients during the coronavirus pandemic. In this case, the FDA is making available an oral outpatient treatment option that can reduce the need for frequent visits to health care facilities," Richard Pazdur, MD, the FDA's top oncology/hematology official, said in a statement. "At this critical time, we continue to focus on providing options to patients with cancer, including regimens that can be taken at home."

Approval was based on two clinical trials showing that intravenous decitabine and the oral decitabine-cedazuridine formulation yielded similar drug concentrations, with patients randomly assigned to the tablet formulation in cycle one followed by intravenous decitabine in cycle two, or to the opposite sequence. All enrolled patients received oral decitabine-cedazuridine starting in cycle three.

Complete response rates in the two trials ranged from 18-21%, with median durations of response of 7.5-8.7 months. Among patients needing red blood cell or platelet transfusions, about half (49-53%) became transfusion independent after 56 days of treatment. Nearly two-thirds of patients that were transfusion independent at baseline remained so at 56 days (63-64%).

Safety outcomes were similar regardless of sequence, according to the FDA, and adverse events occurring in at least 20% of patients included "fatigue, constipation, hemorrhage, myalgia, mucositis, arthralgia, nausea, dyspnea, diarrhea, rash, dizziness, febrile neutropenia, edema, headache, cough, decreased appetite, upper respiratory tract infection, pneumonia, and transaminase increased."

Source: Medpage Today