NEW DELHI: In a landmark advance for thalassemia care, US FDA has approved Mitapivat, the first oral treatment for anaemia in adults with thalassemia, covering both transfusion-dependent and non-transfusion-dependent alpha and beta disease. Clinicians and patient groups in India have welcomed the decision as a potential game changer for a lifelong genetic blood disorder that currently depends largely on repeated blood transfusions and iron chelation. The drug, however, is not yet available in India, as it has not received approval from DCGI. Calling the approval a historic shift, Dr Rahul Bhargava, director and head of hematology and hemato-oncology at Fortis Institute of Blood Disorders, said Mitapivat targets the disease at its cellular core by correcting red-cell metabolism rather than only managing consequences. From a transfusion-medicine perspective, Dr Rishiraj Sinha from AIIMS said adult patients have long borne the clinical and quality-of-life burden of lifelong transfusions and chelation. “The approval of the first oral therapy for both transfusion-dependent and non-transfusion-dependent thalassemia is a meaningful shift in disease management. Beyond improving haemoglobin, an oral option could reduce fatigue, treatment burden and long-term complications, moving care toward a more patient-centred and livable model-though access, affordability and safety monitoring will be critical,” he said.Experts said the next challenge is regulatory clearance and affordability in India. About the AuthorAnuja JaiswalAnuja Jaiswal is a Senior Assistant Editor at The Times of India, with an impressive 18-year career in narrative journalism. She specializes in health and heritage reporting, expertly simplifying complex health information to make it engaging and understandable for readers. Her deep dives into heritage topics are well-researched, resulting in captivating narratives that resonate with her audience. Over the years, she has worked in Chandigarh, Chhattisgarh and West UP, gaining diverse on-ground experience that shapes her storytelling.Read MoreEnd of ArticleFollow Us On Social MediaVideos”Sport Should Be Kept Apart From Politics” Tharoor On Mustafizur RowProbe Reveals Red Fort Blast Module Used ‘Ghost’ SIM Cards For Encrypted ChatsMatter Of Deep Concern: MEA Reacts To US Strikes On Venezuela, Urges Restraint’Lunatic Rulers’: Shashi Tharoor, Singhvi, Owaisi, Other Indian Leaders On US Strikes In VenezuelaIndian Army Raises Bhairav Special Force With Over One Lakh Drone Operatives For Modern WarfareCM Revanth Reddy’s Remarks In Telangana Assembly Sparks Row, BRS Slams ‘Filthy Language’‘If Trump Can Capture Maduro, Why Can’t Modi Bring Back 26/11 Mastermind From Pakistan’: Owaisi‘Avoid All Non-Essential Travel To Venezuela’: India Issues Advisory For Citizens After US Strikes”No Control Over Minority Violence In Bangladesh…”: Former Indian Envoy Slams Yunus Govt“Infamous For His Tour With Anti-National Propaganda”: BJP Rips Rahul Gandhi Over Vietnam Visit123PhotostoriesPCOS beyond periods: How it affects mental health and metabolismMood fixes to outsmart the Monday blues before 9 AM20 minutes visceral fat-burning exercise routine to try at home4 everyday foods that help babies learn to chew naturally10 cheapest markets in Mumbai for street shoppingBeyond the Birkin: 5 times Aishwarya Rai Bachchan proved the ‘Mom Bag’ is the ultimate luxury flex8 common Japanese words and phrases worth learningNayanthara to Tara Sutaria: Decoding first looks of ‘Toxic’s female cast – Who understood the assignment best?Vaginal health: NHS doc debunks popular myths women still believe6 places where the world’s deadliest snakes are found123Hot PicksUS strikes VenezuelaVande Bharat Sleeper TrainPublic holidays January 2026Gold rate todayDelcy RodríguezVivek RamaswamyBank Holidays JanuaryTop TrendingSan Francisco 49ersBrittany MahomesNoah Lyles and Junelle Bromfield Net WorthWayne Gretzky Daughter Net WorthSidney Crosby LifestyleLeBron James vs Stephen Curry Net WorthTom BradyLeBron James WifeCam ThomasCharlie Kirk
NEW DELHI: In a landmark advance for thalassemia care, US FDA has approved Mitapivat, the first oral treatment for anaemia in adults with thalassemia, covering both transfusion-dependent and non-transfusion-dependent alpha and beta disease. Clinicians and patient groups in India have welcomed the decision as a potential game changer for a lifelong genetic blood disorder that currently depends largely on repeated blood transfusions and iron chelation. The drug, however, is not yet available in India, as it has not received approval from DCGI. Calling the approval a historic shift, Dr Rahul Bhargava, director and head of hematology and hemato-oncology at Fortis Institute of Blood Disorders, said Mitapivat targets the disease at its cellular core by correcting red-cell metabolism rather than only managing consequences. From a transfusion-medicine perspective, Dr Rishiraj Sinha from AIIMS said adult patients have long borne the clinical and quality-of-life burden of lifelong transfusions and chelation. “The approval of the first oral therapy for both transfusion-dependent and non-transfusion-dependent thalassemia is a meaningful shift in disease management. Beyond improving haemoglobin, an oral option could reduce fatigue, treatment burden and long-term complications, moving care toward a more patient-centred and livable model-though access, affordability and safety monitoring will be critical,” he said.Experts said the next challenge is regulatory clearance and affordability in India.
