The Food and Drug Administration approved the first drug of its kind for polycythemia vera, a rare blood cancer characterized by the overproduction of red blood cells, in an Aug. 28 announcement that caps a remarkable week of rare disease breakthroughs.
Polycythemia vera affects an estimated 100,000 to 150,000 Americans and carries elevated risks of blood clots, stroke, and transformation to more aggressive cancers, according to the FDA. Prior treatment options were limited, and many patients managed symptoms rather than the underlying disease process.
The “first-in-class” designation means the drug targets a previously untargeted molecular pathway, offering a fundamentally new approach to treating the disease, the FDA said.
The approval is the third first-in-class drug the FDA cleared in a single week, following approvals for metastatic pancreatic cancer on Aug. 26 and dermatomyositis on Aug. 27. The cluster of approvals represents an extraordinary stretch of regulatory activity affecting patient populations historically underserved by pharmaceutical innovation.
For polycythemia vera patients, the new drug offers the prospect of treating the disease itself rather than simply managing symptoms such as elevated blood counts through periodic blood draws, a practice known as phlebotomy. Uncontrolled polycythemia vera can lead to life-threatening complications including heart attack and stroke.
The rare disease designation that polycythemia vera carries under FDA rules provides pharmaceutical developers with incentives including extended market exclusivity, tax credits for development costs, and expedited review pathways.
The drug’s manufacturer, pricing, and specific prescribing information were detailed in the FDA’s full approval letter.