The FDA approves a new treatment option for rare anemia


A rare and potentially deadly product anemia she now has a new treatment option. The Food and Drug Administration announced the approval of the injection of pegcetacoplan (Empaveli) to treat adults paroxysmal nocturnal hemoglobinuria (PNH). Pegcetacoplan is the first HNP treatment to bind to complement the C3 protein, according to the FDA announcement. C3 protein supplement is a key component of host immunity and defense.

Special concern

Due to the risk of serious side effects, the drug is only available through a restricted program under a risk assessment and mitigation strategy (REMS). Serious infections can occur in patients taking pegcetacoplan that can be fatal or fatal if not treated sooner. According to the FDA, REMSs are designed to reinforce drug use behaviors and actions that promote the safe use of this medication and only a few medications require an REMS.

The other most common side effects are injection site reactions, diarrhea, abdominal pain and fatigue.

Pegcetacoplan was approved from a study of 80 patients with PNH and anemia who had been taking eculizumab, a previously approved treatment. During 16 weeks of treatment, patients in the pegcetacoplan group had a mean increase in their hemoglobin of 2.4 g / dL, while patients in the eculizumab group had a mean decrease in their hemoglobin of 1.5 g / dL. dL.

About the disease

HNP is caused by affecting gene mutations red blood cells, causing them to be defective and likely to be destroyed by the patient’s own immune system. The red blood cells of people with these mutations are defective and can be destroyed by the immune system, causing anemia.

Other symptoms include blood clots and destruction of the bone marrow. The disease affects between 1 and 1.5 people per million, with a typical diagnosis between the ages of 35 and 40 and an average survival of only 10 years after diagnosis, according to the FDA.

This article originally appeared on, which is part of the Medscape professional network.

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