According to a publication from BioSpace, the American Food and Drug Administration (FDA) recently approved the Inrebic (fedratinib) for the treatment of certain myelofibrosis patients.
Myelofibrosis is a highly rare form of cancer, characterized by the progressive scarring of marrow and accompanying decline in blood cell production.
You may remember from biology class that red blood cells, along with white blood cells (and platelets) are all made in the bone marrow. As marrow scars, it becomes unable to produce new blood cells to replace those that naturally die off over time. As blood cell counts drop to dangerous levels (anemia), patients may become weak and fatigued. Accompanying platelet count drops can make clotting take longer, making bleeding episodes lengthier and more severe.
Technically, myelofibrosis is a kind of leukemia — a cancer of the blood or blood-forming tissue. Like many pervasive cancers, however, treatment options are mostly limited to alleviating symptoms — regular blood transfusions, chemotherapy, or removal of the spleen (if enlarged).
About Inrebic (Fedratinib)
Inrebic (generic name fedratinib) is the first new treatment for myelofibrosis patients in almost a decade. The developing company, Celgene Corporation, was encouraged to apply for approval of the drug following successful trials involving over 600 total participants — over 450 of whom had myelofibrosis. In a number of trials, fedratinib was found to provide significant therapeutic effect (defined as a reduction in spleen volume equal to or greater than 35%) in roughly 1/3 of patients who received a daily 400mg dose.
Inrebic was approved by the FDA on an priority review basis. Drugs provided through the priority review pipeline take much less time to gain FDA approval — taking only around 6 months of deliberation versus the almost 10 month average. When treating conditions like myelofibrosis, just those four months can be incredibly significant — in some conditions, just four months can literally be life or death.
Additionally, Inrebic received orphan drug designation from the agency. Orphan drug designation exists to benefit drug manufacturers who produce treatments designed for rare diseases — defined as affecting fewer than 200,000 people in the United States. The government supports orphan drug researchers with monetary incentives, research assistance, and a noteworthy 7-year period of monopoly over competing products.
Do you or does anyone you know live with myelofibrosis? What do you think of this exciting development? Share your thoughts with Patient Worthy!