New Delhi, Jan 2 (NationPress) The US Food and Drug Administration (FDA) has made a groundbreaking announcement with the approval of the first oral medication for anaemia in adults suffering from thalassemia. Health experts have termed this a pivotal moment in the management of this inherited blood disorder.

The FDA has sanctioned mitapivat (to be sold as Aqvesme) specifically for treating anaemia in adults diagnosed with either alpha or beta-thalassemia.

This is the inaugural medication designated for both forms of these rare genetic blood disorders, whether or not they depend on transfusions.

Thalassemia patients have traditionally depended on transfusions and chelation therapy for decades, but the introduction of mitapivat signifies a substantial transition in treatment approaches.

“Mitapivat could represent a significant breakthrough in the management of thalassemia,” stated Dr. Satyam Arora, Additional Professor of Transfusion Medicine at the Postgraduate Institute of Child Health (PGICH) in Noida, during an interview with IANS.

“This newly approved drug can revolutionize patient care by allowing management with a single oral pill, which is easily consumable, potentially lowering their blood transfusion needs,” he added.

Experts suggest that beyond merely enhancing haemoglobin levels, an oral treatment could significantly diminish fatigue, lessen the treatment burden, and reduce long-term complications, ushering in a more sustainable, patient-focused care model.

What is Mitapivat?

Mitapivat is classified as a pyruvate kinase activator, being the first of its kind aimed at improving the energy dynamics within red blood cells.

In individuals with thalassemia, red blood cells are often fragile and disintegrate prematurely, resulting in chronic anaemia and a reliance on blood transfusions. The drug's activation of pyruvate kinase promotes cellular energy generation, leading to longer-lasting and more effective red blood cells. Consequently, haemoglobin levels rise, and the frequency of blood transfusions may decrease.

“For the first time, a medication has been developed that directly targets the metabolic processes of red blood cells, tackling the disease at its cellular foundation rather than merely addressing its symptoms. This innovation could dramatically elevate the quality of life for thalassemia patients globally—particularly in India, where the prevalence of the disease is notably high,” explained Dr. Rahul Bhargava, a member of the Delhi Haematology Society.

India holds the title of the thalassemia capital of the world, with every eighth thalassemia patient globally residing in the country, many of whom will need continuous blood transfusions from a young age. Concerns regarding blood availability, complications from transfusions, and iron overload persist.

While the government has made strides in expanding access to bone marrow transplantation through public healthcare systems, most patients still rely on lifelong transfusions.

“The new oral therapy provides optimism not only for transfusion-dependent thalassemia (TDT) patients but also for individuals with non-transfusion-dependent thalassemia (NTDT)—a group that has historically faced limited treatment avenues. By enhancing red blood cell longevity and alleviating anaemia, this medication might help postpone or even avert the shift to transfusion dependence for some patients,” said Bhargava.

Experts expressed their anticipation for the timely availability of this transformative drug in India.

“These are promising times for our thalassemia patients, as well as the blood supply challenges in our country. If the demand for blood among thalassemic individuals decreases, this could substantially enhance blood management in India,” Arora remarked.

“I perceive this as a tremendous beacon of hope for thalassemia patients in India, provided that swift access can be ensured. We will collaborate closely with our medical professionals to assess the drug's efficacy and appropriateness for various patient categories. In summary, this is encouraging news,” concluded Anubha Taneja Mukherjee, Secretary of the Thalassemia Patients Advocacy Group.