Game changer: Experts hail approval of 1st oral pill for adults with thalassemia anaemia

New Delhi, Jan 2 The US Food and Drug Administration’s (FDA) approval of the first-ever oral pill for anaemia in adults with thalassemia marks a historic milestone and can be a game changer in the management of the genetic blood disorder, said health experts on Friday.

The FDA recently approved mitapivat (to be marketed under the brand name Aqvesme) for the treatment of anemia in adults with alpha- or beta-thalassemia.

Mitapivat is the first medication indicated for both transfusion-dependent and non-transfusion-dependent forms of these rare, inherited blood disorders.

While thalassemia patients have, for decades, relied largely on transfusions and chelation therapy, the approval of mitapivat will lead to a meaningful shift in disease management.

“Mitapivat can be a big path-breaking drug for thalassemia management,” Dr. Satyam Arora, Additional Professor of Transfusion Medicine at Postgraduate Institute of Child Health (PGICH), Noida, told IANS.

“This new FDA-approved drug can be a game changer as this will help us manage patients with a single oral pill, which can be easily taken, and their blood transfusion requirements may be reduced,” he added.

According to experts, more than improving haemoglobin levels, an oral option has the potential to reduce fatigue, treatment burden, and long-term complications, while opening the door to more sustainable, patient-centered care.

What is Mitapivat?

Mitapivat is a pyruvate kinase activator, a first-in-class medication designed to improve the energy balance within red blood cells.

In patients with thalassemia, red blood cells are fragile and break down prematurely, leading to chronic anaemia and an ongoing dependence on blood transfusions.

“For the first time, a drug has been developed that directly targets the metabolic pathway of red blood cells, addressing the disease at its cellular core rather than only managing its consequences. This breakthrough has the potential to significantly transform the quality of life for thalassemia patients across the world--and especially in India, where the disease burden remains high,” said haematologist Dr. Rahul Bhargava, Member of the Delhi Haematology Society.

India is the thalassemia capital of the world. Every eighth thalassemia patient in the world lives in India, many of whom will require regular blood transfusions from infancy. Blood availability, transfusion-related complications, and iron overload remain persistent concerns.

While the Government has made significant progress in expanding access to bone marrow transplantation through public healthcare institutions, the majority of patients still depend on lifelong transfusions.

“The new oral therapy offers hope not only for transfusion-dependent thalassemia (TDT) patients but also for those with non-transfusion-dependent thalassemia (NTDT) -- a group that has historically had limited treatment options. By improving red blood cell survival and reducing anaemia, the drug may help delay or even prevent the progression to transfusion dependence in some patients,” Bhargava said.

The experts also expressed hope for the game changer drug to be available soon in India.

“These are very hopeful times for our thalassemia patients as well as the blood requirement burden in our country. If the blood requirement in the thalassemic patient gets reduced, this will significantly help manage the blood requirement in our country more efficiently,” Arora said.

“I see this as a huge ray of hope for patients of thalassemia in India, provided expeditious access can be ensured. We will also work closely with our doctors to find out the efficacy and suitability for various categories of patients. In short, this is good news,” added Anubha Taneja Mukherjee, Secretary of the Thalassemia Patients Advocacy Group.

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