This year, the European Hematology Association (EHA) held its Hybrid Conference starting on June 15; if registered for the conference, you may access the Congress Platform until August 15, 2022. According to a news release from biopharmaceutical companies Sobi and Apellis Pharmaceuticals, Inc. (“Apellis”), the companies shared data from the Phase 3 PRINCE and PEGASUS clinical trials. During the trials, researchers evaluated the safety, efficacy, and tolerability of EMPAVELI (also known as Aspaveli and pegcetacoplan) for adults with paroxysmal nocturnal hemoglobinuria (PNH).

Altogether, 53 previously-untreated patients enrolled in the PRINCE trial. Over 26 weeks, they received either the current standard-of-care for PNH, or 1080mg EMPAVELI twice each week. 80 patients, who were using eculizumab, enrolled in the PEGASUS trial. In the first month, patients received both twice weekly EMPAVELI with eculizumab. Next, in the 16-week period, patients received either EMPAVELI or eculizumab.

In new data analyses, researchers found that:

• EMPAVELI significantly improved quality-of-life (QOL) for previously untreated adults with PNH. In fact, blood counts rose to the point where they were almost considered “normal” (relative to the general population).
• EMPAVELI similarly reduced the risk of thrombosis when compared to eculizumab.
• Using MAIC as an analytical tool, researchers found that EMPAVELI could be more effective in previously untreated patients than the current standards-of-care, such as C5 inhibitors.

EMPAVELI: An Overview

EMPAVELI is an injectable C3 therapy which regulates over-activation within the complement system. Currently, it is approved in Australia, Saudi Arabia, the United States, and both the European Union and United Kingdom. For the latter two, the therapy is called Aspaveli. Your doctor must be enrolled in the REMS program in order to provide you access to this medication. This treatment can increase the risk of serious, life-threatening, or even fatal bacterial infections. Patients must be vaccinated against certain bacteria before beginning EMPAVELI.

Common side effects of EMPAVELI include:

• Infections (including bacterial and viral)
• Fatigue
• Injection-site or hypersensitivity reactions
• Diarrhea
• Abdominal pain or discomfort

Learn more about EMPAVELI.

What is Paroxysmal Nocturnal Hemoglobinuria (PNH)?

Paroxysmal nocturnal hemoglobinuria (PNH) is a rare acquired hematopoietic stem cell (HSC) disorder characterized by red blood cell destruction (hemolysis), blood clots, and impaired or poor bone marrow function. In addition to red blood cells, this disorder also affects white blood cells and platelets. An estimated 30% of PNH diagnoses result from aplastic anemia treatments. PIGA gene mutations, occuring spontaneously, also cause PNH. As abnormal cells proliferate, healthy cells die. PNH typically manifests between ages 35 and 40. While those with mild PNH may survive for decades, the life expectancy following a PNH diagnosis is usually around 10 years. Symptoms can include:

• Dark or bloody urine
• Fatigue
• Headache
• Chest pain
• Difficulty breathing or swallowing
• Hemolytic anemia
• Abnormally pale skin
• General weakness
• Abnormal blood clotting
• Frequent infections
• High heart rate
• Male sexual dysfunction
• Kidney disease