It has been given FDA approval to treat adult patients with pyruvate kinase deficiency who have hemolytic anaemia, and it is also showing promise in treating patients with thalassemia
With the direction and assistance of the Department of Health – Abu Dhabi, two significant international Phase 3 clinical studies for the treatment of thalassemia are about to get underway in Abu Dhabi (DoH).
ENERGIZE and ENERGIZE-T, two adult patient trials, are designed to evaluate the efficiency and safety of Mitapivat, a novel medication.
The clinical trials, which are being carried out in partnership with numerous centres and places throughout the US, Europe, and Asia, may give thalassemia patients in the nation new hope. It is being done in Burjeel Medical City in Abu Dhabi.
The US Food and Drug Administration (FDA) has already approved Agios Pharmaceuticals’ PYRUKYND for the treatment of hemolytic anaemia in people with pyruvate kinase (PK) deficiency (mitapivat). It has, however, also shown promise in preliminary studies for revolutionising the management of thalassemia.
Thalassemia clinical trials
The medicine would be assessed in two kinds of thalassemia with very varied clinical requirements during the Phase 3 programme. The ENERGIZE research is to determine whether Mitapivat can efficiently and safely raise haemoglobin levels in thalassemia patients who are not transfusion-dependent and do not require routine transfusions. Researchers from Burjeel Medical City have demonstrated in prior investigations that low haemoglobin levels can cause considerable morbidity and even death.
ENERGIZE-T seeks to determine whether the same medication can lessen the need for transfusions in thalassemia patients who rely on them.
Clinical trials in Abu Dhabi witnessed a rise of 27% last year, going from 56 trials in 2021 to 71 trials in 2022. Moreover, the UAE was one of the first nations to take part in Covid-19 vaccine clinical trials, hosting the largest Sinopharm vaccine clinical trial, including more than 32,000 participants.
“We are tirelessly working to consolidate Abu Dhabi’s status as a pioneering healthcare destination globally and the region’s centre for research and innovation in life science,” said Dr. Asma Al Mannaei, Executive Director of the Research Center and Innovation at DoH.
While ENERGIZE-T will run for 48 weeks of core treatment periods for primary findings, followed by five-year extensions, the ENERGIZE trials will begin with a standard screening phase and last for 24 weeks.
“If these studies reach their endpoints, this might be a paradigm changer for how the condition is handled today,” said Dr. Khaled Musallam, group chief research officer of Burjeel Holdings. Patients in the UAE and throughout the world may still have unmet needs that a disease-modifying treatment can help with.