FDA approves Agios’ oral leukaemia treatment Tibsovo
pharmafile | July 23, 2018 | News story | Sales and Marketing | FDA, agios, approval, pharmaceuticals, tibsovo
The oral leukaemia treatment Tibsovo (ivosidenib) produced by Massachusetts-based Agios Pharmaceuticals has been approved for treatment of adult patients with relapsed or refractory acute myeloid leukaemia (AML) with a defective IDH1 gene in the United States.
The FDA approved the Cambridge-based drug makers AML pill for use in treating the rapidly progressing bone marrow cancer. The National Cancer Institute at the National Institute of Health estimated that 19,520 people will be diagnosed with AML this year while 10,670 will die from the disease.
The FDA granted the application Fast Track and Priority Review designations while the drug itself received Orphan Drug designation which provides incentives to assist and encourage the development of drugs for rare diseases.
The drug’s effectiveness was studied in a clinical trial of 174 adults with AML who had the defective IDH1 gene. After an average of 8.3 months 32.8% of patients experienced either a complete remission (CR) or a complete remission with partial hematologic recovery (CRh) lasting an average of 8.2 months.
The most common side effects were fatigue, an increase in white blood cells, joint pain, diarrhoea, shortness of breath, swelling in the arms and legs, nausea, pain or sores in the mouth or throat, irregular heartbeat (QT prolongation), rash, fever, cough and constipation.
Dr Richard Pazdur, Director of the FDA’s Oncology Center of Excellence and acting Director of the Office of Hematology and Oncology Products in the FDA’s Center for Drug Evaluation and Research commented: “Tibsovo is a targeted therapy that fills an unmet need for patients with relapsed or refractory AML who have an IDH1 mutation. The use of Tibsovo is associated with a complete remission in some patients and a reduction in the need for both red cell and platelet transfusions.”
Louis Goss
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