Paroxysmal nocturnal hemoglobinuria (PNH) prematurely destroys the bodies red blood cells (hemolysis). It can cause severe anemia, kidney disease, fatigue, as well as other symptoms. Up until now, there has been only one approved treatment for the condition (eculizumab). But just recently, Ultomiris (ravulizumab-cwvz) received approval by the FDA as a treatment for adults with the PNH.
This approval is significant for PNH patients as it would be a less burdensome treatment option. Too often, patients with rare conditions have no choice when it comes to their treatment. This new approval gives patients more of a say in their own treatment regime.
The approval of Ultomiris is also currently being considered by regulatory agencies in Japan and the European Union. Hopefully, we will see approval in these locations soon.
You can read more about this new approval and the research that went into it here.