FDA/CDC

FDA approves oral therapy for myelodysplastic syndromes, CMML


 

The Food and Drug Administration has approved Inqovi (decitabine and cedazuridine tablets, Astex Pharmaceuticals) to treat adults with myelodysplastic syndromes (MDS) or chronic myelomonocytic leukemia (CMML). The agency’s action gives physicians and patients a new treatment option – an oral-formulation MDS and CMML medication that can be taken at home.

Approval of the tablets could obviate the need for some patients to come to healthcare settings for intravenous therapy, a consideration that goes beyond patient convenience. “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, director of the FDA’s Oncology Center of Excellence, stated in a news release.

“At this critical time, we continue to focus on providing options to patients with cancer, including regimens that can be taken at home,” added Dr. Pazdur, who is also acting director of the office of oncologic diseases in the FDA’s Center for Drug Evaluation and Research.

Inqovi received an Orphan Drug designation and a Priority Review from the agency.

The FDA based the new formulation approval on clinical trials that showed patients taking Inqovi had similar drug concentrations, compared with others receiving intravenous decitabine.

The two therapies also had similar safety profiles. Fatigue, constipation, hemorrhage, muscle pain, mucositis, arthralgia, nausea, and fever with low white blood cell count were common side effects reported in people taking Inqovi. The agency noted that Inqovi can cause fetal harm, and that both male and female patients of reproductive age are advised to use effective contraception.

In the clinical trials, approximately half of the patients formerly dependent on transfusions no longer required them during an 8-week period.

Inqovi is taken as one tablet by mouth once daily for 5 consecutive days of each 28-day cycle.

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