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FDA approves the first C3 inhibitor for adults with serious rare blood disease

Release time:

2021-05-21 13:46

FFROM approvesthe first C3 inhibitor for adults with serious rare blood disease

On May 14th, 2021, Apellis Pharmaceuticals Inc. announced that the U.S. Food and Drug Administration (FDA) has approved a new injection treatment, EMPAVELI™ (pegcetacoplan), for adults with paroxysmal nocturnal hemoglobinuria (PNH). It is the first and only PNH medication that targets C3, the central protein in the complement system.

Paroxysmal nocturnal hemoglobinuria, PNH in short, is a rare, acquired, life-threatening blood disorder. The key features of the disease are red blood cell destruction, blood clots, and impaired bone marrow function. PNH results from gene mutations that affect red blood cells. Patients with mutatedPIGA on the X chromosome have abnormal hematopoietic stem cells which produce defective red blood cells. The destructive blood cells can be detected and destroyed by the immune system, which results in hemolytic anemia, or other symptoms, such as red discoloration of the urine. The prevalence rate is about 1 to 1.5 people per million. Individuals are typically diagnosed around ages 35 to 40, and the PNH patients have an average survival of 8-15 years after diagnosis. Nevertheless, around 25% of the patients may live more than 25 years with only minor symptoms.

As the first and FDA-approved targeted C3 therapy, EMPAVELI (pegcetacoplan) has better effectiveness than C5 inhibitors. It can treat PNH adults who have never undergone PNH treatment or the patients who previously took C5 inhibitors, Soliris® (eculizumab), or Ultomiris® (ravulizumab). The approval of Empaveli was based on data from a multi-center, randomized, head-to-head Phase III study (ClinicalTrials.gov: NCT03500549) which compared the efficacy and safety of Empaveli to Solirisin 80 adults with PNH. The patients were divided into two groups which were randomly assigned to received either Empaveli 1,080 mg/20mL twice weekly or their current dose of Soliris for 16 weeks.The severity of anemia was compared in the two treatment groups which had the average 8.7 g/dL hemoglobin at baseline. Normal hemoglobin values in adult men and women are more than 14 g/dL and 12 g/dL respectively. After the 16-week treatment, patients in the Empaveli group averagely increased 2.4 g/dL of their hemoglobin. Meanwhile, patients in the Soliris group averagely decreased 1.5 g/dL of their hemoglobin.Moreover, 85% of the Empaveli -treated patients were transfusion free compared to 15% of the Soliris-treated patientsoThis 16 weeks.

Empaveli is available only through a restricted program under a Risk Evaluation and Mitigation Strategy (Empaveli REMS;https://empavelirems.com/#Main) due to the risk of serious infections. In addition, the most common side effects are injection site reactions, viral/ microbial infections, diarrhea, abdominal pain, respiratory tract infection, and fatigue.

It must be said that Empaveli is a significant milestone that boosts the development of C3 therapies for rare diseases. The approval of EMPAVELI not only gives more treatment options of PNH but also brings new promise for the PNH community.

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