What are the indications for Soliris?

Soliris generally refers to eculizumab, an innovative drug for rare diseases, commonly formulated as an injection. It was officially launched in China in 2022. Eculizumab has been approved for three indications: refractory generalized myasthenia gravis in patients positive for anti-acetylcholine receptor antibodies, paroxysmal nocturnal hemoglobinuria (PNH), and atypical hemolytic uremic syndrome (aHUS). The details are as follows:

1. Refractory generalized myasthenia gravis in patients positive for anti-acetylcholine receptor antibodies

This medication is indicated only for adult patients. This condition is an autoimmune disorder caused by impaired transmission at the neuromuscular junction, which can lead to loss of muscle function and severe weakness.

2. Paroxysmal nocturnal hemoglobinuria (PNH)

This medication can be used in both adults and children. PNH is a non-malignant clonal disease caused by acquired somatic mutations in the PIG-A gene in one or more hematopoietic stem cells. The PIG-A gene mutation leads to abnormal synthesis of glycosylphosphatidylinositol (GPI), resulting in the loss of a group of membrane proteins anchored to the cell surface via GPI. Clinically, it mainly manifests as chronic intravascular hemolysis, bone marrow failure, and recurrent thrombosis.

3. Atypical hemolytic uremic syndrome (aHUS)

This medication can be used in both adults and children. aHUS is a complement-mediated disorder. Mutations in genes encoding complement regulatory proteins such as factor H, membrane cofactor protein, and intrinsic serum complement components may all contribute to disease development. The condition tends to relapse frequently, carries a poor prognosis, and may result in death.

The above are the three approved indications for eculizumab. Patients are advised to seek timely medical attention when experiencing symptoms and to undergo standardized treatment under the guidance of a physician, which may help promote recovery.