Novartis Partners with Ghana to Treat SCD, Offering Hydroxyurea and Opening Adakveo Trial

Ghana is working with Novartis to make hydroxyurea treatment available to people with sickle cell disease, and to open a clinical trial in the African country testing the company’s biologic SCD therapy Adakveo (crizanlizumab), recently approved in United States.

The five-year, public-private partnership was formed in January among the Ministry of Health of Ghana, Ghana Health Service, the Sickle Cell Foundation of Ghana, and the pharmaceutical company.

An estimated 15,000 babies are born with sickle cell disease in Ghana each year, and the country’s leaders are hoping to use a holistic approach to battle SCD, improving disease diagnosis and screening, and patient treatment.

Novartis has already delivered more than 20,000 hydroxyurea treatments being used at 11 centers across Ghana with trained personnel, and is expected to cover patients’ needs for up to a year, officials said in a press release. The company has committed to sending 60,000 additional treatments to be used at others centers the country is opening by the close of 2019.

Ghana is the first African nation to commit to offering the international standard of care to its residents.

“I am proud of this bold partnership, and it is my hope that, through this collaboration, we will help ease the pain and improve the lives of people living with sickle cell disease in our country,” said Alhaji Mahamudu Bawumia, ME and vice president of the Republic of Ghana.

“Our collective goal is to reimagine what the future could look like for people with sickle cell disease. Where children do not need to miss out on school or be singled out. Where young adults can have equal opportunity for employment. And where families can flourish and continue to be the bedrock of our civil society,” he added.

“In a nutshell, we will make every effort to ‘normalize’ sickle cell disease within the Ghanaian society.”

Commonly used to lower the frequency of pain crises and need for blood transfusions in SCD patients, hydroxyurea was approved last fall by Ghana’s health regulatory agency, granting marketing rights to Novartis.

The company has also committed to developing a child-friendly version of this treatment.

Hydroxyurea works by increasing levels of a type of hemoglobin called hemoglobin F, or fetal hemoglobin, which helps to prevent anemia and frequent pain crises.

Clinical trials of Adakveo, approved by the the U.S. Food and Drug Administration (FDA) to treat SCD in patients ages 16 and above, are planned to open in Ghana next year, Novartis said in a company press release. A similar trial of crizanlizumab will also open in Kenya.

Adakveo was approved based on results from the yearlong Phase 2 SUSTAIN trial (NCT01895361) in 198 SCD patients with a history of frequent vaso-occlusive crises. Data showed treatment, given each month by intravenous infusion at a 5 mg/kg dose, lowered the annual rate of these pain crises by 45% compared to placebo, and the number of days spent in a hospital each year.  

About 80% of SCD patients worldwide are born in sub-Saharan Africa, where it’s reported that more than half die before age 5 due to preventable disease complications.

“Novartis is deeply committed to reimagining medicine for patients in Ghana and across Africa,” said Vas Narasimhan, MD, Novartis CEO. “Building on our long heritage of addressing malaria and leprosy in Africa, we’re excited to help improve the diagnosis and treatment of people with sickle cell here in Ghana.”

Through the partnership, Ghana and Novartis plan to collaborate on field testing and implementing SCD treatment guidelines, and establishing regional centers of excellence.

They will also work to ensure that newborn screening is in place at those centers, and to use digital technology to monitor and assess patient registration, report real-time data, and to help ensure the safe, widespread roll out of SCD treatments.