BHUBANESWAR: The New Year seems to have ushered in a major breakthrough for thalassaemia patients with the US Food and Drug Administration (FDA) approving the first-ever oral pill to treat adults suffering from the inherited blood disorder.
The drug Mitapivat has demonstrated significant ability to reduce dependence on blood transfusions. Mitapivat is a pyruvate kinase (PK) activator. Since PK enzyme is crucial for energy production in red blood cells, Mitapivat has been designed to improve the energy balance within red blood cells and help patients with inherited hemolytic anaemias.
According to haematologists and bone-marrow transplant physicians, this drug could transform long-term disease management. Thalassaemia is a common inherited haemoglobin disorder caused by mutations in the alpha, beta or delta globin genes, resulting in impaired haemoglobin synthesis and ineffective erythropoiesis. It is a major public health concern in eastern India, Odisha being one of the high burden states for such haemoglobinopathies, including sickle cell disease.
Despite advanced treatment options like bone marrow transplantation and emerging gene therapies, patients continue to rely on regular blood transfusions due to limited access, high cost and donor unavailability.
Secretary of the Indian College of Haematology (ICH) and former head of clinical haematology department at SCBMCH Prof RK Jena described the FDA approval as a potential game-changer and said this drug could significantly improve patient outcomes. “Frequent blood transfusions in patients often lead to iron overload and damage to vital organs such as the liver, heart and endocrine system. If an oral medication can meaningfully reduce transfusion requirements, nothing can be better than this,” he said.
Prof Jena, who was part of India’s first-ever study on thalidomide as a potent haemoglobin inducer, suggested that Mitapivat be introduced alongside thalidomide to achieve better therapeutic outcomes. Thalidomide, known for its immunomodulatory properties, has shown encouraging results in reducing transfusion dependence by increasing foetal haemoglobin production.
“The safety and efficacy of thalidomide have been evaluated in 404 patients aged between two and 18 years with beta thalassaemia major or intermedia. It offers a convenient, effective and affordable option for patients who are not eligible for allogeneic stem cell transplantation,” he said.
The Indian College of Haematology has already recommended the use of thalidomide at a dose of two to four milligrams per kg of body weight for thalassaemia patients, he said.