According to a story from BioSpace, the biopharmaceutical company Bristol Myers Squibb Canada recently announced that its medication fedratinib (marketed as INREBIC®) has been approved by Health Canada as a treatment for myelofibrosis, a form of rare bone marrow cancer. The drug is specifically intended to treat spleen enlargement and the associated symptoms, which common occurs as a result of myelofibrosis. It is approved for patients with intermediate or high risk disease, including disease associated with thrombocythemia or polycythemia vera.
Myelofibrosis is considered a rare type of bone marrow cancer. The disease is characterized by the excessive accumulation of abnormal stem cells in the bone marrow which trigger a process called fibrosis, or scarring. Over time, the bone marrow is replaced with scar tissue. While the exact cause of myelofibrosis is not known, genetic mutations affecting the MPL, JAK2, and CALR genes are known risk factors. Symptoms of myelofibrosis include enlarged spleen, anemia, shortness of breath, easy bruising and bleeding, greater risk of infection, bone pain, gout, fatigue, weight and appetite loss, and increased blood cell volume. As a cancer that affects stem cells, stem cell transplant can cure the disease. However, this process carries many significant risks. Other forms of treatment are symptomatic and supportive and do not alter the course of myelofibrosis. There is a dire need for safer and more effective therapies for the disease. To learn more about myelofibrosis, click here.
INREBIC is the first therapy approved in almost ten years that has demonstrated the ability reduce spleen size in myelofibrosis patients. As there is a clear need for more effective therapeutic options in this illness, the approval of a new treatment has the potential to improve quality of life for these patients.
This drug is classified as an inhibitor of the janus kinase (JAK). INREBIC is intended to be taken once per day. The drug was evaluated in the phase 3 clinical trial that included a total of 289 patients. In this trial, treatment with this therapy was able to fulfill the primary endpoint of 35 percent or more reduction in spleen size.