FDA Approves At-Home Therapy to Treat Myelodysplastic Syndromes

July 11, 2020

A new oral therapy approved by the FDA will provide patients with myelodysplastic syndromes, who typically visit a health care facility for intravenous treatment, with an oral at-home option that can be beneficial during the coronavirus pandemic.

The FDA has approved decitabine and cedazuridine (Inqovi), oral tablets to treat adults with myelodysplastic syndromes (MDS) and chronic myelomonocytic leukemia (CMML). Previously, patients with MDS received treatment intravenously at a health care facility.

The drug, from Astex Pharmaceuticals, was also approved by Health Canada, and it has been submitted for review with Australia’s Therapeutic Goods Administration. The therapy was submitted and reviewed through the FDA’s Project Orbis, an initiative of the FDA Oncology Center of Excellence that provides framework for therapies to be submitted and reviewed among international partners.

“The FDA remains committed to providing additional treatments to patients during the coronavirus pandemic.,” Richard Pazdur, MD, director of the FDA’s Oncology Center of Excellence and acting director of the Office of Oncologic Diseases in the FDA’s Center for Drug Evaluation and Research, said in a statement. “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.”

Decitabine is a hypomethylating agent and cedazuridine is a cytidine deaminase inhibitor. The approval was based on the ASCERTAIN phase 3 study, as well as supporting phase 1 and 2 studies. The trials evaluated the equivalence between the oral therapy and intravenous decitabine. The studies also showed that about half of the patients who were dependent on transfusion no longer needed transfusions during an 8-week period.

The safety profile of the oral therapy was similar to intravenous decitabine. The most common adverse effects (≥20%) 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 increase. The most common serious adverse events (>5%) were febrile neutropenia (30%), pneumonia (14%), and sepsis (13%).

“Intravenous or subcutaneous administered hypomethylating agents have been the cornerstone for the treatment of patients with MDS and CMML since the mid-2000s,” Guillermo Garcia-Manero, MD, professor and chief of section of myelodysplastic syndromes, Department of Leukemia at The University of Texas MD Anderson Cancer Center, and principal investigator of ASCERTAIN, said in a statement. “The FDA’s approval of Inqovi builds on the proven therapeutic utility of hypomethylating agents in these diseases and offers a new orally administered option that offers patients an alternative to five consecutive days of IV infusions every month during a treatment period that can extend to several months.”