New FDA Approval of Ultomiris for Paroxysmal Nocturnal Hemoglobinuria

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.


Ultomiris is a long-acting C5 complement inhibitor. The investigation of its efficacy for PNH patients was examined in the largest phase 3 program ever conducted for the condition. This program included two separate phase 3 clinical trials.
In the first trial, 246 patients were examined and every single endpoint confirmed that Ultomiris was noninferior to standard therapy. The second trial of 191 patients showcased the same results. The drug was administered just once every 8 weeks which is a significantly longer period than the typical 2 week dose of eculizumab. The reduction of the number of treatments needed could itself significantly improve the quality of life of patients and their families.
The most common adverse event reported in the study of this drug was headache. However, it is important to note that Ultomiris does have a warning regarding its associated risk with meningococcal infections and sepsis. That said, no such infections occurred during the trials. The FDA simply states that patients should be immunized with the vaccine at least two weeks before starting treatment of Ultomiris unless there is a circumstance where the risk of infection outweighs the risk of PNH.

Looking forward

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.

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