EU Approves Aspaveli to Treat Paroxysmal Nocturnal Hemoglobinuria

According to a recent article, Apellis Pharmaceuticals and Swedish Orphan Biovitrum AB recently announced the European Commission (EU) has approved Aspaveli to treat adults with paroxysmal nocturnal hemoglobinuria (PNH).

Paroxysmal Nocturnal Hemoglobinuria (PNH)

Paroxysmal nocturnal hemoglobinuria (PNH) is an acquired hematopoietic stem cell disorder that affects all three types of blood cells: red, white and platelets. The disease can be fatal, with those surviving approximately 10 years after diagnosis. For more mild cases, individuals can survive decades after being diagnosed.

PNH destroys red blood cells and causes abnormalities in platelets. It can occur at any time, to males and females equally, and up to 30% of cases are a result of aplastic anemia treatment. The approximate median age of diagnosis is 35-40, and it affects one to two people per million.


  • Red blood cell destruction
  • Dark or bloody urine
  • Fatigue
  • Increased heart rate
  • Chest pain
  • Headache
  • Difficulty breathing
  • Abdomen contractions
  • Difficulty swallowing
  • Sexual dysfunction (men)
  • Kidney disease


Aspaveli is the first and the only targeted C3 therapy used to treat patients with PNH. It works by regulating the excessive activation of the complement cascade (a part of the immune system), which can lead to the start and progression of a number of serious diseases. The therapy is used for patients who are anemic after treatment that has a C5 inhibitor for at least three months. 72% of patients who are treated with a C5 inhibitor remain anemic, even though there have been efforts to improve hemolytic activity with the treatment.

The treatment was approved according to a PEGASUS Phase 3 study. In this study, Aspaveli was compared with eculizumab in terms of efficacy and safety. Patients in the study were adults with PNH who has persistent anemia despite being treated with eculizumab. The patients were examined after 16 weeks and the treatment was shown to be safe and effective.

A Significant Approval

The EU approving Aspaveli is important for patients dealing with PNH since the symptoms of the disease impact the quality of a patient’s life greatly. In addition, since the other treatments already available do not help with a patient’s anemia, patients require blood transfusions often. Therefore, Aspaveli being approved is a sign of hope to improve the treatment options for patients with PNH.

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