The US Food and Drug Administration (FDA) has approved mitapivat (brand name Aqvesme) for treating anaemia in adults with thalassaemia, working by improving red blood cell energy. The twice-daily pill can be taken by patients who need regular blood transfusions or those who do not. Experts say it could transform disease management in high-burden countries like India.
Thalassaemia is an inherited blood disorder that impairs the body's ability to produce haemoglobin and healthy red blood cells. The US FDA has recently approved mitapivat, branded as Aqvesme, as the first oral medication to treat anaemia in adults with this condition. The drug improves the energy balance inside red blood cells, allowing them to survive longer and transport oxygen more effectively.
Dr Rahul Bhargava, director and head of haematology at Fortis Institute of Blood Disorders in Gurugram, describes it as a milestone: "For the first time, a drug has been developed that directly addresses the disease at its cellular core rather than only managing its consequences. This can significantly transform the quality of life for thalassaemia patients across the world—and especially in India, where the disease burden remains high."
Clinical trials, including ENERGIZE and ENERGIZE-T, have demonstrated increases in haemoglobin levels and reductions in transfusion needs. The therapy benefits both transfusion-dependent thalassaemia (TDT) patients and those with non-transfusion-dependent thalassaemia (NTDT). Dr Anupam Sachdeva, director of paediatric haematology and oncology at Sir Ganga Ram Hospital in New Delhi, notes that the ACTIVATE-Kids trial has confirmed its safety and efficacy in children.
In India, around 8,000 babies are born with thalassaemia major each year, with most relying on lifelong transfusions. The drug is not yet available here, pending approval from the Drugs Controller General of India (DCGI). Patient advocate Anubha Taneja Mukherjee, secretary of the Thalassaemia Patients Advocacy Group, calls it "a ray of hope if expeditious access can be ensured. We also need to find out the efficacy and suitability for various categories of patients."
This treatment is more targeted than blood transfusions and iron chelation therapy, and potentially more accessible than curative bone marrow transplants. Once introduced in India, it could be supported by government programs, easing the economic, physical, and emotional burden on patients, families, and the healthcare system.
