FDA Approves New Therapy for Rare Blood Cancers that Can Be Taken at Home

The U.S. Food and Drug Administration approved Astex Pharmaceuticals’ Inqovi tablets for treatment of adult patients with the rare blood cancers myelodysplastic syndromes and chronic myelomonocytic leukemia.

Inqovi is the first and only orally administered hypomethylating agent for the treatment of adults with intermediate and high-risk myelodysplastic syndromes (MDS) including chronic myelomonocytic leukemia (CMML), two blood malignancies.

MDS comprise a heterogeneous group of hematopoietic stem cell disorders characterized by dysplastic changes in myeloid, erythroid, and megakaryocytic progenitor cells, and associated with cytopenias, such as infections and bleeding, affecting one or more of the three lineages. Incidence of MDS in the United States is estimated to be 10,000 cases per year, with an estimated prevalence of 60,000 to 170,000 patients. MDS may evolve into acute myeloid leukemia in one-third of patients, and the prognosis is poor.

CMML is a clonal hematopoietic malignancy characterized by accumulation of abnormal monocytes in the bone marrow and in blood. The incidence of CMML in the U.S. is approximately 1,100 new cases per year, and CMML may transform into AML in 15 percent to 30 percent of patients.

The hypomethylating agents decitabine and azacitidine are effective treatments are FDA-approved for the treatment of intermediate and high-risk MDS and CMML. These agents are administered by IV infusion, or by large-volume subcutaneous injections, which must be done at a health care facility.

Inqovi (decitabine and cedazuridine) is taken as one tablet by mouth once daily for five consecutive days of each 28-day cycle. The approval of Inqovi represents an important advance in treatment options for patients with MDS, as they can now treat themselves at home.

“Until now, patients with intermediate and high-risk MDS and CMML have not had an approved, orally administered hypomethylating agent option for treatment of their disease,” said Mohammad Azab, president and chief medical officer of Astex Pharmaceuticals. “The Inqovi clinical program was designed to deliver an oral alternative to IV decitabine based on comparative decitabine exposure data in the clinical trials, and to assess Inqovi’s safety and efficacy profile.”

The approval was based on clinical trial results that showed similar drug concentrations between intravenous decitabine and Inqovi. Additionally, about half of the patients who were formerly dependent on transfusions were able to no longer require transfusions during an 8-week period. The safety profile of Inqovi was also similar to intravenous decitabine.

Some common side effects of Inqovi included fatigue, constipation, hemorrhage, muscle pain, mucositis (mouth sores), arthralgia (joint pain), nausea, and fever with low white blood cell count. Inqovi can cause fetal harm, and both male and female patients of reproductive age are advised to use effective contraception.

The FDA had previous granted Astex, a subsidiary of Japanese pharmaceutical Otsuka, Orphan Drug status and Priority Review for its new drug application. The FDA also collaborated with international agency counterparts on the review of the application as part of Project Orbis.

Taiho Pharmaceutical, also a subsidiary of Otsuka, will commercialize Inqovia in the United States and Canada.

Photo: Mohammad Azab, president and chief medical officer of Astex Pharmaceuticals

Author: Rare Daily Staff