Khaled Musallam: UAE Approves Pyrukynd for Both Alpha and Beta Thalassemia
Khaled Musallam, Group Chief Research Officer at Burjeel Holdings, shared on LinkedIn:
”Some welcome and long awaited good news — and a reminder that even in challenging times, progress continues.
I am truly delighted to share that Pyrukynd (mitapivat) has now been approved in the UAE for adult patients with both alpha and beta thalassemia, including transfusion-dependent and non-transfusion-dependent forms.
This approval follows the landmark global ENERGIZE and ENERGIZE-T trials, which we were honored to participate in.
We celebrated when the trials met their primary endpoints. We celebrated when the data were presented in plenary sessions at EHA and ASH.
We celebrated again when the results were published in The Lancet.
But today marks something even more meaningful — the translation of rigorous science into real access for patients in the UAE.
Mitapivat represents the first oral disease-modifying therapy for thalassemia, addressing both anemia and transfusion burden across subtypes.
Bringing such an innovative therapy to our patients is the most important outcome we could have hoped for.
My sincere gratitude to the leadership of Burjeel Holdings for supporting what was our first global clinical trial within the group — a defining step in our research journey.
I am incredibly proud of our research and clinical teams at the Thalassemia and Sickle Cell Center and the Center for Research on Rare Blood Disorders (CR-RBD) at Burjeel Cancer Institute | BCI, BMC | Burjeel Medical City, whose dedication made this milestone possible.
This achievement would not have been possible without the exceptional support of the Department of Health Abu Dhabi.”
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