Possible Treatment for Paroxysmal Nocturnal Hemoglobinuria Submitted for FDA Approval

According to a story from Business Wire, the company Alexion Pharmaceuticals recently announced that it has submitted a Biologics License Application (BLA) to the FDA for the approval of one of the company’s investigational products, ALXN1210. This therapy is intended for the treatment of patients with paroxysmal nocturnal hemoglobinuria. This submission carries a priority review voucher, which would allow the FDA to review the drug for a period of eight months instead of the standard twelve months.
Paroxysmal nocturnal hemoglobinuria (PNH) is a rare blood disease that causes the complement system, a component of the nervous system, to begin destroying red blood cells. This is due to a defective protein on the surface of the blood cells that, if functioning properly, would normally protect them from destruction. Paroxysmal nocturnal hemoglobinuria is not genetically caused; the defect is instead acquired. It may appear on its own or be the result of other disorders such as aplastic anemia. The most well known symptom is red urine, but not all patients have it. Other symptoms include anemia, abdominal pain, erectile dysfunction, difficulty swallowing, and painful swallowing. Blood clots are also common are the most dangerous complication. To learn more about paroxysmal nocturnal hemoglobinuria, click here.

The best available treatment that is currently approved for PNH is called eculizumab. Although it is quite effective, it has attracted significant controversy because it is extremely expensive. A year of treatment costs $440,000. Another weakness of eculizumab is that it greatly increases the risk of meningococcal infection; the risk increases by at least 1000 times compared to the average population. This requires patients to get a vaccination before treatment and take measures to prevent exposure to infection during treatment.

In Phase 3 trials, ALXN1210 was compared side by side to the earlier medication. In all measures, the new drug was found to be superior or equal in effectiveness and safety in comparison to eculizumab. ALXN1210 also received Orphan Drug designation from both the FDA and the European Medicines Agency (EMA). Alexion plans to submit approval applications for the new drug in the E.U. and in Japan before the end of the year.

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