Gene Therapy for Sickle Cell Disease Remains Unaffordable in Africa Despite US, UK Approvals
Approved gene-editing therapies for sickle cell disease priced at $2.2 million remain out of reach in Africa, where 80% of the world's cases occur, as Uganda launches mandatory newborn screening.
As Uganda rolls out mandatory nationwide screening of newborns for sickle cell disease this month, a gene therapy celebrated in the United States and Europe remains financially out of reach for most patients in Africa. According to the World Health Organization, about 515,000 babies are born each year with sickle cell disease, a lifelong, inherited blood disorder. Around 80 per cent of cases occur in Sub-Saharan Africa, where it is a leading cause of deaths among young children.
In Uganda, the Ministry of Health launched a policy this month (9 February) requiring all babies born in health facilities to be screened for the disease at birth, free of charge, in a bid to reduce preventable deaths. Official figures show an estimated 20,000 Ugandan children are born with the condition each year, with up to 80 per cent dying before the age of five, largely due to delayed diagnosis and preventable complications.
While African governments expand early detection and basic treatment, high-income countries have been adopting the first approved gene-editing therapy designed to cure sickle cell disease. The United States Food and Drug Administration approved Casgevy (exagamglogene autotemcel) for patients aged 12 years and over in December 2023, hailing it a "milestone" treatment. The UK Medicines and Healthcare products Regulatory Agency approved the therapy weeks earlier and it was authorised for use in the National Health Service in England in January this year.
The FDA also approved Lyfgenia, a cell-based gene therapy manufactured by Bluebird Bio, though it comes with a warning of possible cancer risks.
Sickle cell disease is caused by a mutation in the gene that produces haemoglobin, the protein that carries oxygen in red blood cells. The defect causes cells to become rigid and "sickle" shaped, leading to severe pain, organ damage and shortened life expectancy.
Casgevy works by taking a patient's blood stem cells, editing them in the laboratory using CRISPR gene-editing technology to reactivate healthy haemoglobin production, and then reinfusing them into the body after chemotherapy. If successful, the patient begins producing functional red blood cells. Clinical trials showed the therapy dramatically reduced severe pain episodes in most patients treated.
However, the breakthrough comes with a staggering cost, with Casgevy priced at around US$2.2 million in the United States. The manufacturer, Vertex, did not respond to questions about pricing for low- and middle-income countries. Bluebird Bio also didn't provide a response ahead of publication.
The price tag for gene therapy makes it largely symbolic in Nigeria, a country where many families can't even afford basic medicines. Nigeria carries the world's largest burden of sickle cell disease, with an estimated 150,000 babies born with the condition each year. Millions more live with the disorder and many lack access to consistent treatment.
For decades, treatment in Africa has focused on managing symptoms through pain relief, infection prevention, blood transfusions and, in limited cases, bone marrow transplants—previously the only established cure. Nigeria has established three bone marrow transplant centres offering this treatment. However, costs range from US$50,000 to over US$200,000, making that also unaffordable for most families.
Trust is another barrier. Even when procedures are available at home, many prefer to seek treatment abroad if they can afford it.
Gene therapy's advantage over bone marrow transplantation is that it does not require a donor—an important consideration for African patients, who face lower donor match rates globally.
Gene therapy prices reflect both the complexity of the science and the infrastructure required to deliver it. Most gene therapy today relies on viral vectors and requires highly specialised, good manufacturing practice facilities that cost millions of dollars to build, so companies sell these therapies at an extremely high price to recover the costs incurred.
In the United States, SCD affects approximately 100,000 people as of 2024, yet more than 90% of patients are non-Hispanic Black or African American. Patients with SCD often experience vasoocclusive episodes (VOEs), hemolytic anemia, organ damage, and failure, thus reducing their expected life span by 20 years when compared with the national average.
Other FDA-approved therapies for SCD include L-glutamine (Endari; Emmaus Medical, Inc), voxelotor (Oxbryta; Global Blood Therapeutics), and crizanlizumab (Adakveo; Novartis), which alleviate VOEs. A more recent therapy for treating SCD, isoquercetin, shows promise in targeting thromboinflammation in adults with SCD.