▎ WuXi AppTec content team editor
Recently, a thrombopoietin (TPO) receptor agonist, romidadine for injection (former name: romidastim for injection), was approved by the State Drug Administration of China (NMPA) for marketing for primary immune thrombocytopenia (ITP).
Screenshot source: NMPA official website
Rare autoimmune diseases
Immune thrombocytopenia is a rare and severe autoimmune disease that is a blood disorder. People with immune thrombocytopenia, whose immune systems attack and destroy their own platelets, which play an active role in blood clotting and wound healing.
Immune thrombocytopenia is characterized by a low platelet count in the blood, which can lead to serious bleeding events or complications and even death.
The common symptoms of adult immune thrombocytopenia are repeated mucocutaneous bleeding, such as petechiae, purpura, ecchymosis, and difficulty in stopping bleeding after trauma; nosebleeds, bleeding gums, menorrhagia, etc. are also common; severe visceral bleeding is more rare.
At the same time, the condition of patients with immune thrombocytopenia may also be suddenly aggravated by infection and other reasons, with extensive and severe bleeding from the skin and mucous membranes and internal organs.
Fatigue is another common symptom of immune thrombocytopenia. In addition, excessive bleeding or prolonged menorrhagia can also lead to hemorrhagic anemia.
According to the 9th edition of "Internal Medicine" data, the incidence of immune thrombocytopenia in Chinese adults is 5-10/100,000 people, with similar incidence rates in men and women; the incidence of women in childbearing age is higher than that of men; the elderly over 60 years old are high-incidence groups, the incidence rate is twice that of people under 60 years old, and the risk of bleeding increases with age.
Image credit: 123RF
Thrombopoietin receptor agonists
At present, there is no cure or radical cure for immune thrombocytopenia, and the purpose of first- or second-line treatment is to raise the patient's platelet count to a safe level and reduce mortality.
First-line therapy for immune thrombocytopenia is the use of glucocorticoids or intravenous immunoglobulins; second-line therapy consists mainly of various immunologic agents, thrombopoietin receptor agonists, and splenectomy.
Lopsin is a recombinant protein that belongs to the thrombopoietin receptor agonist, which, by binding to the thrombopoietin receptor, stimulates the intracellular transcriptional pathway, resulting in increased thrombopoiesis.
Public information shows that roppestine is produced in E. coli through recombinant DNA technology, which can simulate the effect of the body's natural thrombopoietin, thereby improving platelet counts. When lopstidine enters the body and binds to thrombopoietin receptors, it promotes the growth of bone marrow megakaryocyte colony-forming units (CFU-Meg) and leads to increased thrombocytosis through the JAK2 and STAT5 kinase pathways.
In addition, the mechanism of action of lopespressin by stimulating thrombopoietin receptors is very different from the drugs commonly used to increase platelet counts by inhibiting platelet destruction. In 2009, Roppsting was also selected as the "Best Biotechnology Product" by the Galen Prize, known as the "Nobel Prize of the Pharmaceutical Industry".
At present, lopsudine has been approved for marketing in dozens of countries and regions around the world, such as the United States, the European Union, Canada, Australia, etc., and the approved indications include adult patients with immune thrombocytopenia who are poorly treated with other drugs or surgeries, and pediatric patients aged 1 year and above who persist in immune thrombocytopenia for at least 6 months and do not respond to corticosteroids, immunoglobulins or splenectomy.
We expect that with the approval of roppestine for marketing in China, more patients with immune thrombocytopenia in China will benefit."