Bahrain has become the second country in the world to approve Casgevy, a CRISPR-based therapy for two inherited blood disorders: sickle cell disease and the transfusion-dependent form of beta-thalassemia. The Daily Tribune reported the decision on December 3, 2023. It is also the first approval anywhere in the Middle East.
The treatment, known generically as exagamglogene autotemcel, was from Vertex Pharmaceuticals together with CRISPR Therapeutics. The only regulator to clear it earlier was the UK’s MHRA, and Bahrain’s approval followed that authorization. The Daily Tribune said the therapy went through successful clinical trials in several countries, among them the UK, the US, France, Italy and Germany.
How the therapy works
Bahrain’s National Health Regulatory Authority (NHRA) described the procedure in plain terms. Stem cells are taken from an eligible patient, and a healthy copy of those cells is put back into the body. LocalBH, which covers Bahraini news and events, wrote that the NHRA reviewed the treatment carefully before confirming approval.
Who gets it first
No patient list has been published yet. The regulator said officials were still working out which groups and which patients qualify.
The decision was tied to the directives of Crown Prince and Prime Minister Prince Salman bin Hamad Al Khalifa, who has made good healthcare for citizens a priority. Dr. Ahmed Alansari, the NHRA’s chief executive, said the approval “marks a significant turning point in the array of treatments on offer to Bahrainis.”
Only the UK had moved first.
Sources
- Bahrain makes medical history (The Daily Tribune)
- Bahrain Becomes the 1st Country in the Middle East to Approve Revolutionary Treatment (LocalBH)
