NDA to be Submitted for Potential Paroxysmal Nocturnal Hemoglobinuria Treatment

According to a story from globenewswire.com, the biopharmaceutical company Apellis Pharmaceuticals, Inc. has announced that it is currently making plans to submit a New Drug Application (NDA) to the US Food and Drug Administration (FDA). This application will be for pegcetacoplan, a medication in development for the treatment of paroxysmal nocturnal hemoglobinuria, a rare disease. The company plans to submit the application in the second half of this year. Apellis is prioritizing the development of targeted C3 therapies.

About Paroxysmal Nocturnal Hemoglobinuria

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.

Hope for Approval

Apellis plans to submit the application using data from a phase 3 study in which pegcetacoplan was compared side by side to eculizumab, a current standard treatment for paroxysmal nocturnal hemoglobinuria. In this trial, the therapy met the primary endpoint by demonstrating statistically significant advantages in therapeutic effect over eculizumab. In addition, the safety profile of pegcetacoplan was not significantly different from eculizumab.

The company is also aiming to get the drug approved in the European market as well, and discussions for marketing authorization are currently ongoing for the region.


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