US FDA Approves Innovative Therapy for Blood Cancer

The FDA has given its approval for Tibsovo (ivosidenib) to be used in the treatment of adult patients suffering from relapsed or refractory myelodysplastic syndromes (MDS) who possess an is citrate dehydrogenase-1 (IDH1) mutation as identified through an FDA-approved test. The approval of this indication marks the first approved targeted therapy for this specific purpose. In addition to its approval of the Abbott RealTime IDH1 Assay as a companion diagnostic, the agency has also granted its approval for the same assay to be used for selecting R/R MDS patients with an IDH1 mutation.

The approval of Tibsovo has been granted to Servier Pharmaceuticals LLC by the US FDA, while the RealTime IDH1 Assay has received approval from Abbott Laboratories, as per pharmabiz. Myelodysplastic syndromes (MDS) are a type of blood cancers that are uncommon. They can develop when there are mutations in the progenitor cells of the bone marrow, which are responsible for forming blood. These mutations can result in a lack of enough healthy blood cells. In the United States, the number of individuals living with MDS is estimated to be between 60,000 and 170,000. On a global scale, there are approximately 87,000 new cases of MDS reported every year.

Approximately 3.6 percent of individuals with MDS exhibit an IDH1 mutation. According to Richard Pazdur, M.D., the 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, today's approval signifies a significant progression in the treatment of rare blood cancers. This advancement specifically benefits patients with relapsed or refractory MDS who have an IDH1 mutation. We are dedicated to fostering scientific innovation and facilitating the progress of safe and effective novel therapies for patients suffering from rare cancers. This commitment is evident through the FDA’s Oncology Center of Excellence Rare Cancers Programme.