FDA Approves First-Ever Drug to Treat Specific Rare Blood Disease
The Food and Drug Administration (FDA) has approved Genentech’s Zelboraf, as a treatment for patients diagnosed with a specific mutation of that disease.
BioSpace writes, the drug was approved specifically for the treatment of ECD patients with BRAF V600 mutation. Originating in the bone marrow, the disease is a rare, serious blood disease characterised by the abnormal multiplication of certain white blood cells called histiocytes, which can invade normal tissues and organs in the body.
According to Genentech, more than 50% of people with ECD have BRAF V600 mutation-positive disease. President of ECD Global Alliance, Kathleen Brewer, said, “The Erdheim-Chester disease community is very encouraged by this first FDA-approved treatment for ECD, bringing new hope to patients and their families. This new treatment option shows that meaningful breakthroughs can occur rapidly when patients, families, research physicians, industry and the FDA work together to help patients.”
Sandra Horning, Genentech’s chief medical officer believes that the FDA approval marks the first time people living with ECD have an approved treatment option.
The expanded approval for the disease took place after data was published from the Phase II VE-Basket study. The specific design of the study provided for a faster collection of data that lead to the accelerated development of the drug, Genentech explained.
The approval is a demonstration of how drug makers can “apply knowledge of the underlying genetic characteristics of certain malignancies to other cancers,” concluded Richard Pazdur, director of the FDA’s Oncology Centre of Excellence.