The Health Regulatory Authority of the Kingdom of Bahrain has approved the gene editing therapy Kasgebi (exagamglogen autotemsel) to treat patients with sickle cell disease (SCD) and transfusion-dependent beta-thalassemia (TDT).
The decision makes Bahrain, a small island nation in the Persian Gulf, the second country to recognize Kasgevi. The treatment was approved in the UK last month for adults and children aged 12 and over who are eligible for a stem cell transplant but do not have a donor. SCD patients require repeated vaso-occlusive crises (VOCs).
Details regarding treatment eligibility for patients in Bahrain were not available.
“The approval of Kasgebi for use in Bahrain marks a significant turning point in the range of treatments being offered to Bahrainis. It is a testament to our unwavering commitment to improving the lives of our people while providing them with the highest quality of health care and welfare,” said Ahmed Alansari, CEO of the country’s National Health Regulation. authorities said in a press release.
Gene-editing sickle cell therapy is effective in restoring fetal hemoglobin production
Bahrain’s Minister of Health, Jaleela Jawad, added that the approval “demonstrates Bahrain’s progressive approach to healthcare and its determination to remain at the forefront of scientific progress.”
Casgevy, also known as exa-cel, is under priority review for approval in the United States, with a decision expected this week. Applications for approval of the treatment in the European Union and Saudi Arabia are also under review.
Sickle cell is caused by a mutation that leads to the production of an abnormal form of adult hemoglobin, a protein that red blood cells use to transport oxygen through the bloodstream. Abnormal proteins can form clumps within red blood cells, changing their shape and making circulation in blood vessels more difficult and causing VOCs and other disease symptoms.
Kasgeby is designed to increase the production of fetal hemoglobin. Fetal hemoglobin is an alternative form of protein that is normally produced only during early fetal development and is more effective at transporting oxygen than adult hemoglobin. This is expected to reduce hemoglobin aggregation.
This treatment involves collecting and manipulating the patient’s hematopoietic stem cells. Hematopoietic stem cells are located in the bone marrow and are responsible for producing new blood cells. The harvested cells are manipulated using CRISPR gene editing tools to increase fetal hemoglobin production. The modified cells can then be returned to the patient through a stem cell transplant to generate new blood cells that can produce fetal hemoglobin.
29 of 30 SCD patients treated in clinical trials did not consume VOCs for 1 year
Regulatory approval of Kasgevy is primarily supported by data from ongoing Phase 1/2/3 clinical trials. One is called CLIMB-121 (NCT03745287) for his SCD patients and the other is called CLIMB-111 (NCT03655678) for his TDT patients. Participants who complete either study will be able to participate in her expansion study, called CLIMB-131 (NCT04208529), which will collect long-term safety data for this treatment.
CLIMB-121, scheduled to conclude in October 2024, is evaluating the safety and efficacy of a single dose of Kasugebi in patients with severe SCD who had at least two VOCs in the two years prior to enrollment. .
According to trial data provided by the U.S. Food and Drug Administration, 29 of 30 evaluable patients cleared VOCs within 1 year of treatment with Kasugevy, meeting the primary goal of CLIMB-121.