BAHRAIN has approved a new medication that can be used to help treat Sickle Cell Disease (SCD) patients from the age of five.
Endari, having been given the green light by the US Food and Drug Administration, has also now been approved for marketing in Bahrain by the National Health Regulatory Authority (NHRA).
NHRA chief executive Dr Mariam Al Jalahma revealed that Bahrain was the second country in the GCC to register Endari, a medication used to reduce the acute complications of Sickle Cell Disease (SCD) in adults and children.
“NHRA is keen to expedite the registration process for innovative drugs to ensure they are available in Bahrain,” she said. “This is especially when it comes to emergency use, rare diseases or for the treatment of cancer.”
The haemoglobin of SCD sufferers is abnormal causing the red blood cells to become hard and sticky and take the appearance of a c-shaped farm tool called a sickle.
The sickle cells die early which results in a constant shortage of red blood cells.
Patients are at risk of a variety of acute and chronic health issues, including infection, stroke and sickle cell crises can result in death. There is no known cure for the genetic disorder.
Endari is an oral L-glutamine therapy by Emmaus Life Sciences for sickle cell anaemia and sickle beta-thalassaemia, two types of SCD.
“We are grateful that Bahrain officials have seen fit to make Endari available to sickle cell patients in the country,” said Emmaus Life Sciences chief executive Dr Yutaka Niihara in an official statement published online.
“It furthers our mission to afford increased access to Endari in the Middle East and North Africa region, as well as in India, with their large and underserved sickle cell disease patient populations.”
According to online research, Endari works to increase the amount of free glutamine circulating in the blood and sickle cells can take up this free glutamine and use it to generate anti-oxidant molecules.
These new anti-oxidants can help neutralise the oxidative stress in sickle cells and allow them to regain the flexibility necessary to travel through blood vessels and capillaries, carrying oxygen to tissues throughout the body.
The FDA decision to approve Endari made it the first approved treatment for children with SCD, and the first new treatment for adults with the disease in almost 20 years.
“SCD patients require a multi-faceted treatment approach which could include surgery, psychological care, social support and medication and over the past 30 years there has only been one medicine for SCD globally with some pharmaceutical companies refusing to develop another one,” claimed Bahrain Sickle Cell Society chairman Zakariya Al Kadhem.
“Bahrain has spared no efforts to ensure that top quality health care is provided to SCD patients and this new medicine is another advanced step in that direction to provide the best possible treatment available.
“Medication will allow patients to continue their life without continued doctor visits which then allows them to flourish and lead a productive life.”
He added that another SCD medication is in the clinical trials phase.
As recently reported in the GDN, the kingdom is making all efforts to increase the average age of sickle cell patients by a further 10 years after successfully raising their life expectancy to 65 this year.
Mr Al Kadhem revealed that the country was making rapid strides with only 10 per cent of the around 9,000 patients requiring hospitalisation for the disease. Meanwhile, 90pc of the sufferers only require treatment for other illnesses, similar to healthy individuals.
He was speaking during a special celebration held at the Hereditary Blood Disorder Centre at Salmaniya Medical Complex to commemorate World Sickle Cell Day, observed every year on June 19.
reem@gdnmedia.bh