Farjah Algahtani: A New Era in the Treatment of Transfusion-Dependent Thalassaemia
Farjah Algahtani, Director Epidiomolgy and Public health Research Chair at King Saud University, shared a post on LinkedIn about a recent article by Maria Domenica Cappellini et al, published in The Lancet, adding:
“A new era in the treatment of transfusion dependent thalassaemia
I am pleased to highlight the recently published ENERGIZE T Phase 3 trial in The Lancet, evaluating mitapivat, an oral allosteric activator of pyruvate kinase, in adults with transfusion dependent α and β thalassaemia.
For many years, reducing transfusion burden through an effective oral disease modifying therapy has remained a major unmet need for patients with thalassaemia.
Key findings from the newly published ENERGIZE T trial:
- 30 percent of patients receiving mitapivat achieved a transfusion reduction response, compared with 13percent receiving placebo.
- The difference was statistically significant (p equal 0.0003).
- Mitapivat demonstrated a generally favourable benefit risk profile.
These important findings support mitapivat as the first oral disease modifying therapy for adults with transfusion dependent α or β thalassaemia, providing a new treatment option to reduce transfusion burden.
Of particular importance for Saudi Arabia, the Saudi Food and Drug Authority (SFDA) approved mitapivat (Pyrukynd) last year, marking an important milestone in expanding access to innovative therapies for patients with thalassaemia.
The publication of the ENERGIZE T study in The Lancet now provides robust Phase 3 evidence supporting this important therapeutic advancement.
I am particularly happy and proud to have been part of this important international clinical trial, representing King Saud University and contributing to global efforts to advance the care of patients with thalassaemia.
It is a privilege to collaborate with international colleagues and contribute to clinical research that has the potential to make a meaningful difference in the lives of our patients.
This is an exciting time for the thalassaemia community and another important step towards transforming the treatment landscape for our patients.
Congratulations to all investigators, patients, and everyone involved in making this important study possible.”
Title: Efficacy and safety of mitapivat in adults with transfusion-dependent α-thalassaemia or β-thalassaemia (ENERGIZE-T): a double-blind, randomised, multicentre, placebo-controlled, phase 3 trial
Authors: Maria Domenica Cappellini, Prof Sujit Sheth, Ali Taher, Hanny Al-Samkari, Ali Bülent Antmen, David Beneitez, Giovanna Cannas, Prof Thomas Coates, Lauren Czapla, Jayme L Dahlin, Jeremie H Estepp, Elizabeth Feenstra, Pencho Georgiev, Sarah Gheuens, Gavrielle M Price, Andreas Glenthøj, Prof Khaled M Musallam, Kareem Osman, Prof John B Porter, Hui Shao, Katrin Uhlig, Eduard J van Beers, Prof Vip Viprakasit, Prof Kevin H M Kuo, Prof Antonis Kattamis
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