In a landmark development for global blood disorder care, the US Food and Drug Administration (FDA) has approved the first-ever oral pill to treat anaemia in adults with thalassemia. Health experts say the decision could significantly reduce dependence on lifelong blood transfusions for patients with this inherited condition.
FDA Approves First Oral Drug for Thalassemia-Related Anaemia
FDA Approval Marks a Historic First
The FDA has approved mitapivat, to be marketed under the brand name Aqvesme, for adults with alpha- or beta-thalassemia. This is the first medicine authorised for both:
- Transfusion-dependent thalassemia
- Non-transfusion-dependent thalassemia
Until now, treatment largely relied on regular blood transfusions and iron chelation therapy, often beginning in early childhood.
How Mitapivat Works
Mitapivat is a first-in-class pyruvate kinase activator that targets the underlying metabolic defect inside red blood cells.
In thalassemia:
- Red blood cells are fragile
- They break down prematurely
- Chronic anaemia develops
Mitapivat improves cellular energy production, helping red blood cells survive longer, increasing haemoglobin levels, and reducing the need for frequent transfusions.
Expert Views on Clinical Impact
Calling the approval a path-breaking advance, Satyam Arora of the Postgraduate Institute of Child Health, Noida, said the drug offers the possibility of managing thalassemia with a single oral pill.
Haematologist Rahul Bhargava noted that this is the first therapy to directly address the disease at the cellular level, rather than only treating its complications, with potential to transform patient quality of life.
Significance for India’s Disease Burden
India carries one of the highest global burdens of thalassemia, with nearly one-eighth of the world’s patients living in the country. Experts believe the new oral therapy could:
- Reduce blood demand
- Lower transfusion-related complications
- Improve long-term outcomes
Patient advocacy groups have expressed hope for early access in India, calling the approval a major step toward sustainable and patient-centred care.
Exam-Oriented Key Facts
- Mitapivat is the first oral drug approved for anaemia in adult thalassemia
- It acts as a pyruvate kinase activator
- Approved for both transfusion-dependent and non-dependent thalassemia
- Approval granted by the US Food and Drug Administration
- May reduce dependence on blood transfusions
Question & Answer
Q1. Which drug has been approved as the first oral treatment for anaemia in adult thalassemia patients?
(a) Deferasirox
(b) Hydroxyurea
(c) Mitapivat
(d) Erythropoietin
Answer: Mitapivat
Q2. Mitapivat works by activating which enzyme in red blood cells?
(a) Hexokinase
(b) Pyruvate kinase
(c) Lactate dehydrogenase
(d) Catalase
Answer: Pyruvate kinase
Q3. The approval of the first oral thalassemia drug was granted by which authority?
(a) WHO
(b) EMA
(c) CDSCO
(d) US Food and Drug Administration
Answer: US Food and Drug Administration
Q4. Mitapivat has been approved for which forms of thalassemia?
(a) Only alpha-thalassemia
(b) Only beta-thalassemia
(c) Only transfusion-dependent thalassemia
(d) Both transfusion-dependent and non-dependent thalassemia
Answer: Both transfusion-dependent and non-dependent thalassemia
Q5. Why is mitapivat considered a game changer in thalassemia treatment?
(a) It replaces surgery
(b) It cures the disease completely
(c) It targets the disease at the cellular level
(d) It eliminates genetic mutations
Answer: It targets the disease at the cellular level
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