US approves first oral medicine to treat anemia caused by thalassemia

The US Food and Drug Administration (FDA) has approved mitapivat, the first oral medicine to treat anemia caused by thalassemia—a major breakthrough for patients who have long depended on lifelong blood transfusions to survive.

The drug has been approved for adults with alpha- or beta-thalassemia, including both transfusion-dependent patients who need regular blood transfusions and those with non–transfusion-dependent forms of the disease. This makes mitapivat the first medicine of its kind to treat anemia across the full spectrum of thalassemia severity.

Calling the approval a landmark moment, Brian Goff, CEO of Agios Pharmaceuticals, said in a statement the decision brings an innovative oral treatment to people living with a serious and lifelong genetic blood disorder.

HOW MITAPIVAT WORKS

Mitapivat works differently from traditional treatments. It is a pyruvate kinase activator, which helps red blood cells produce energy more efficiently, allowing them to survive longer in the bloodstream. This leads to higher haemoglobin levels, reduced fatigue, and in some cases, fewer transfusions.

The FDA’s approval was based on results from two large phase 3 trials—ENERGIZE and ENERGIZE-T—which together included more than 450 patients worldwide. The studies showed that patients taking mitapivat had improved haemoglobin levels and better energy levels. Importantly, some transfusion-dependent patients were able to reduce or even stop blood transfusions.

SAFETY AND AVAILABILITY

Mitapivat carries a warning for possible liver injury, meaning patients will require regular liver tests, especially during the first six months of treatment. Because of this risk, the drug will be available only through a special safety



monitoring programme.

Agios Pharmaceuticals expects the medicine to be available in the US by January 2026.

WHAT DOES THIS MEAN FOR INDIA?

At present, mitapivat is not yet available in India. While US FDA approval is a major global milestone, the drug will still need clearance from Indian regulatory authorities before it can be marketed in the country. Experts, however, are optimistic, given India’s high burden of thalassemia and the lack of effective long-term treatment options.

Dr Rahul Bhargava, Director and Head of Hematology, Hemato-Oncology and Stem Cell Transplant at Fortis Memorial Research Institute, Gurugram, called the decision significant.

“Until now, treatment options for thalassemia were limited to blood transfusions, bone marrow transplant and gene therapy,” he said. “This is a very welcome move. An oral drug that reduces transfusion dependence can improve survival and quality of life, as every unit of blood adds to iron overload.”

He added that if the Government of India is able to procure the drug and make it accessible, it could be a boon for millions of thalassemia patients, especially in low- and middle-income settings.

WHY THIS APPROVAL MATTERS

The FDA’s decision marks a global turning point in thalassemia care. For the first time, patients have access to an oral, disease-modifying drug that targets the underlying problem in red blood cells, not just the symptoms.

For countries like India, where thousands of children are born with thalassemia each year, this breakthrough offers hope for fewer transfusions, lower complications, and a better quality of life, moving care beyond lifelong support to targeted, science-driven treatment.