Novartis Announces New Pediatric Indication Approval for Eltrombopag in China

On May 20, Novartis announced that a new indication for eltrombopag olamine tablets had been approved in China. It is used to treat chronic immune (idiopathic) thrombocytopenia in children aged 6-11 who have previously responded poorly to treatments such as glucocorticoids and immunoglobulins. This makes it the only thrombopoietin receptor agonist (TPO-RA) drug currently available in China for treating chronic immune (idiopathic) thrombocytopenia in children.

Immune Thrombocytopenia (ITP), previously also known as idiopathic thrombocytopenic purpura, is an acquired autoimmune bleeding disorder characterized primarily by isolated peripheral blood thrombocytopenia without a clear cause. It accounts for approximately one-third of all bleeding disorders. According to statistics, the incidence rate of ITP in China's adult population is 5-10 per 100,000 people, with a higher prevalence among individuals over 60 years old, women of childbearing age, and children. Among these groups, the lives of pediatric patients and their guardians are often disrupted by the disease, leading to anxiety, restricted mobility, and a decline in quality of life. Prolonged use of glucocorticoids in first-line treatment significantly impacts children’s growth and development. A "platelet-increasing drug" suitable for long-term management of pediatric patients is crucial for improving the quality of life for children with ITP.

Currently, glucocorticoids and intravenous immunoglobulin are the first-line therapies for ITP. Drugs that promote platelet production, including recombinant human thrombopoietin (rhTPO), eltrombopag, and rituximab, are second-line treatments. Eltrombopag ethanolamine tablets, a non-peptide thrombopoietin receptor (TPO-R) agonist developed by Novartis, are used to treat chronic ITP patients who have had poor responses to previous treatments such as glucocorticoids and immunoglobulin. The drug was approved by the FDA in November 2008 and became the first oral medication for ITP treatment. In 2021, its global sales reached $2.016 billion.

Novartis' eltrombopag olamine tablets were approved in China in 2017 for adult ITP patients who had previously responded poorly to treatments such as glucocorticoids and immunoglobulins. In 2018, the indication was expanded to include children aged 12 years and above, and this time it has been further extended to cover children aged 6 to 11 years.

In recent years, China has paid high attention to the prevention and treatment of pediatric blood diseases and malignant tumors, introducing a series of policies to safeguard the health of pediatric patients. In 2019, ITP was included as one of the ten diseases in the first batch of national medical treatment and healthcare management for pediatric diseases.