Currently, C5 inhibitors such as eculizumab and ravulizumab are considered the standard-of-care for people living with paroxysmal nocturnal hemoglobinuria (PNH). Unfortunately, not all patients respond well to these therapies. How can we improve treatment efficacy and provide an additional therapeutic option? One answer: Empaveli (pegcetacoplan). Approved for use in the United States in 2021, Empaveli is a C3 inhibitor, targeting the C3 protein in the complement cascade. According to a news release from Sobi North America, Empaveli was recently approved in Canada for the treatment of PNH. In fact, it is the first targeted C3 therapy to be approved in the country.
While C5 inhibitors reduce hemolysis (destruction of red blood cells), they do not prevent anemia in around 70% of patients. Researchers evaluated the efficacy of eculizumab in comparison to pegcetacoplan in the Phase 3 PEGASUS study. 80 people enrolled. 85% of those taking Empaveli no longer needed red blood cell transfusions to combat anemia, compared to just 15% of those taking eculizumab. Empaveli was relatively safe and well-tolerated. Adverse reactions included viral and respiratory tract infections, abdominal pain, diarrhea, injection site reactions, and fatigue.
The data from the PEGASUS study was used to help approve the treatment in Canada. Learn more about Empaveli.
An Understanding of Paroxysmal Nocturnal Hemoglobinuria (PNH)
PIGA gene mutations cause paroxysmal nocturnal hemoglobinuria, a rare acquired hematopoietic stem cell disorder. The gene mutation creates PNH cells which survive immune system attacks while healthy cells die. These mutations occur spontaneously. PNH affects red and white blood cells, as well as platelets. On average, the prognosis for PNH is around 10 years following diagnosis. However, with treatment or milder cases, survival rate can be much longer. PNH typically affects 1-2 out of every 1 million people, and manifests between the ages of 30-40. Up to 30% of cases result following treatment for aplastic anemia. Symptoms and characteristics can include:
- Hemolytic anemia
- Platelet abnormalities
- Abnormal blood clotting
- Dark or bloody urine
- Fatigue
- Abnormally pale skin
- Increased heart rate
- General weakness
- Increased risk of infections and leukemia
- Shortness of breath and/or difficulty breathing
- Chest pain
- Abdominal contractions
- Difficulty swallowing
- Male sexual dysfunction
- Kidney disease
