Possible Treatment for Paroxysmal Nocturnal Hemoglobinuria Recommended for Marketing Approval in the EU

According to a story from Market Screener, the biopharmaceutical company Alexion Pharmaceuticals has recently announced some encouraging news regarding the status for ravulizumab (marketed as ULTOMIRIS) in the EU. The Committee for Medicinal Products for Human Use (CHMP), which is part of the European Medicines Agency (EMA) has released an opinion that is in favor of approval of ULTOMIRIS in the EU as a treatment for paroxysmal nocturnal hemoglobinuria (PNH).

About Paroxysmal Nocturnal Hemoglobinuria (PNH)

Paroxysmal nocturnal hemoglobinuria (PNH) is a rare blood disease. This life-threatening illness is most characterized by the destruction of red blood cells by a part of the innate immune system called the complement system. Paroxysmal nocturnal hemoglobinuria is an acquired disease, meaning that it is not caused by genetic abnormalities. It can appear on its own or alongside other blood diseases, such as myelodysplastic syndromes or aplastic anemia. The disease occurs because of a defect in a protein that appears on the surface of red blood cells called DAF. Symptoms of the disease include red urine (especially in the morning), palpitations, blood clots, fatigue, shortness of breath, pain when swallowing, abdominal pain, and erectile dysfunction. Blood clots are a primary cause of death for patients with paroxysmal nocturnal hemoglobinuria. This disease is best treated with either eculizumab or, more recently, ravulizumab where it is available. To learn more about paroxysmal nocturnal hemoglobinuria, click here.

A New Standard of Care?

The recommendation for CHMP comes following encouraging results from two Phase 3 clinical trials. These studies are the largest related to paroxsymal nocturnal hemoglobinuria ever conducted and included a total of 440 patients. These trials compared ULTOMIRIS to SOLIRIS (eculizumab), a drug that was previously developed by Alexion for treating the disease and first entered the market in 2007. The primary advantage of ULTOMIRIS is that the drug does not have to be taken as frequently; their mechanism of action is similar, however. The data indicated that the efficacy of these two drugs is comparable.

ULTOMIRIS was first approved in the US in December 2018. Following the release of the CHMP opinion, the EMA is expected to make a final decision on the approval within the next two months.


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