Abu Dhabi to start clinical trials to develop 'game-changing' new thalassaemia treatment

Abu Dhabi will start clinical trials to test a new drug that could transform the way thalassaemia is being treated.

Burjeel Medical City will test the effectiveness and safety of new drug Mitapivat, which has shown promise as a treatment for the condition.

Thalassaemia is an inherited blood disorder and one of the most common genetic illnesses in the UAE.

It occurs when the body doesn't make enough of a protein called haemoglobin. Severe cases require frequent blood transfusions.

Stem cell or bone marrow transplants are the only current cures for thalassaemia.

Mitapivat is a US and EU-approved drug to manage pyruvate kinase deficiency.

It has recently demonstrated potential in earlier trials for transforming the treatment of thalassaemia.

Abu Dhabi will take part in the phase 3 trials — Energise and Energise-T — which will test Mitapivat's effectiveness on adults with thalassaemia.

The trials will evaluate the drug in two types of thalassaemia with relatively different clinical needs.

The Energise study aims to evaluate whether Mitapivat can effectively and safely improve haemoglobin levels in patients who do not require regular transfusions.

The Energise-T study aims to evaluate whether the same drug can decrease transfusion requirements in patients who regularly receive transfusions.

Dr Asma Al Mannaei, executive director of the Research Centre and Innovation at Abu Dhabi Department of Health, said the emirate's regulator is “working on cementing Abu Dhabi’s position as a pioneering healthcare destination globally and the region’s hub for research and innovation in life science”.

“Abu Dhabi was able to play a pivotal role in developing medicines and conducting clinical trials with several international partners,” she said.

“Today, we are witnessing the launch of new clinical trials so that we continue our efforts as we aim to further improve healthcare services in Abu Dhabi, and helping all members of our community, and across the globe.”

The UAE was one of the first countries to participate in clinical trials of Covid-19 vaccines with more than 32,000 volunteers for the Chinese Sinopharm inoculation.

Dr Khaled Musallam, group chief research officer or Burjeel Holdings, said Abu Dhabi was “now at the forefront of leading-edge innovations”.

“If these studies meet their endpoints, this could be a game changer to how the disease is being treated today,” he said.

“A disease-modifying therapy can address persisting unmet needs of patients in the UAE and around the world.”

The Energise trials will start with a typical screening period and run for 24 weeks, while Energise-T will run for 48 weeks, with core treatment periods for primary results, followed by five-year extensions.

The trials, which will be conducted at Burjeel Medical City in collaboration with the US as well as several centres in the EU and Asia, will include clinical, radiological and laboratory assessments.