Emirati man first in UAE to receive innovative treatment for rare liver disease

Abu Dhabi: A 21-year-old Emirati man has become the first patient in the UAE to receive Givosiran, an innovative treatment for a rare liver disease, at a hospital in Abu Dhabi.

Supported by the Department of Health – Abu Dhabi (DoH), the injection was administered at Burjeel Medical City (BMC) to treat a rare genetic disorder that causes a buildup of toxic metabolites in the liver.

Muhammad, the patient treated at BMC, was diagnosed with acute intermittent hepatic porphyria (AIP), a rare condition that affects only five in one million people.

Dr Niyas Khalid, a specialist in internal medicine at BMC, noted that AIP is a fatal condition, with Givosiran being the only available treatment.

“AIP is a rare genetic disease, and patients suffer from attacks of severe abdominal pain, nausea, and vomiting,” Dr Khalid told Gulf News.

AIP, caused by a defective enzyme, leads to the accumulation of toxic metabolites in the liver. These metabolites trigger symptoms such as neuropsychiatric disorders and can result in life-threatening complications, including renal failure, paralysis, liver failure, and liver cancer.

“Up to 60 per cent of patients can progress to renal failure and develop liver cancer. Other symptoms include high blood pressure, seizures, fatigue, tremors, tachycardia, and palpitations. It causes neuropsychiatric derangement, with patients becoming anxious and experiencing low energy.”

Normalcy in life

Dr Khalid said that Muhammad had been suffering for the past five years, undergoing multiple investigations and tests without a definitive diagnosis.

“When he first came to me, I suspected this disease and initially conducted a urine test, which revealed elevated levels of toxic metabolites. To confirm the diagnosis, we proceeded with a genetic test, which provided definitive confirmation.”

After years of waking up in severe pain and enduring other symptoms like fatigue and weight loss, Muhammad is now experiencing some semblance of a normal life.

“He used to wake up with pain every day. This drug has transformed his life. From being constantly sick and feeling low, he is now a confident man. The number of attacks has reduced as well,” Dr Khalid said.

Approved drugs

Givosiran is now included on the list of approved drugs in the UAE. The DoH’s Research and Innovation Centre’s multidisciplinary team evaluated, coded and documented the medication with an identification code, which enables healthcare facilities in the emirate to efficiently provide the medication.

Dr Asma Al Mannaei, Executive Director of the Health Life Science Sector at the DoH, confirmed that the drug has been added to the health insurance network.

“Administered as a monthly injection, this innovative treatment uses small interference RNA technology to effectively reduce the levels of toxic metabolites in the body,” she said.

The medical team at BMC continues to monitor Muhammad’s progress, aiming for Givosiran to significantly improve his quality of life and prevent further complications.