New drug for sickle cell disease gets the nod

Wednesday July 19 2017
sickle cell

A procession to mark the World Sickle Cell Day in Mombasa on Kenya’s Coast. PHOTO FILE | NMG

By Elizabeth Merab

The US Food and Drug Administration (FDA) has approved a new drug, Endari (also called L-glutamine oral powder), to help reduce complications associated with sickle cell disease.

The drug is the first treatment approved for patients with sickle cell disease in almost 20 years, and is recommended for patients from the age of five years upwards.

“Until now, only one other drug was approved for patients living with this serious, debilitating condition,” said the director of the FDA’s Oncology Centre of Excellence, Richard Pazdur.

Hydroxyurea has been used to manage the condition for more than two decades.

READ: Scientists discover permanent cure for sickle cell anaemia

Pathologists in Kenya have welcomed the news, saying it presents promising results for patient care.


“It is quite exciting that we now have a drug we can add to our stable,” said Dr Valerie Magutu, adding that although it is not a cure, it presents hope for future treatment of sickle cell disease.

The safety and efficacy of Endari were studied in a randomised trial of patients with sickle cell disease aged five to 58 years, who had two or more painful crises within the 12 months prior to enrollment in the trial.

The participants were then randomly given treatment with the new drug or a placebo and evaluated over the course of 48 weeks.

From the study, patients who were treated with Endari had reduced frequency and length of hospital visits for sickle cell pain crises compared with those on the placebo.

READ: Teenager's sickle cell reversed with world-first therapy

The drug also cut the need for blood transfusions, which is common among sickle cell patients due to reduced level of haemoglobin during crises.

“On average, compared with patients who received a placebo, those on Endari had fewer hospitalisations for sickle cell pain and spent fewer days in the hospital,” the scientists noted.

Patients who received Endari also had fewer occurrences of acute chest syndrome (a life-threatening complication of sickle cell disease) compared with patients who received a placebo (8.6 per cent vs 23.1 per cent).

Commonly reported side effects of the drug included constipation, nausea, and headache.

READ: Sickle cell anaemia deaths are ‘avoidable’ — study

Approximately five per cent of the world’s population carries trait genes for haemoglobin disorders, mainly sickle-cell disease and thalassaemia. In many sub-Saharan African countries like Kenya, accurate data about the disease is unavailable.

However, it is estimated that in some parts of Kenya, the prevalence of the disease can be as high as 35 per cent (in coastal Kenya) and 24 per cent in (Western Kenya. These are areas prone to malaria.

“We are seeing a shift in the disease patterns due to intermarriage and movement of people,” explained Prof Jessie Githanga, a specialist pathologist.

However, Dr Magutu said that the lack of affordable diagnostic systems prevents early diagnosis of the disease.

“The standard test, Haemoglobin electrophoresis (referred to as HB electrophoresis) can cost anything between $25 and $70,” she said.

The other reason for the late diagnosis is because children never exhibit sickle cell related problems in the first six months after birth.

This is because in babies have a special haemoglobin in their blood called foetal haemoglobin, that masks the sickle haemoglobin.

Related:Uganda now steps up fight against sickle cell