Promising new sickle cell disease medicine wins FDA approval
After a recent decision by the FDA a better day could be coming for those living with sickle cell disease.
This week, Switzerland-based pharmaceutical company Novartis announced in a press release that the US Food and Drug Administrationhad approved Adakveo a medicine they believe will help, “reduce the frequency of vaso-occlusive crises (VOCs), or pain crises, in adult and pediatric patients aged 16 years and older with sickle cell disease.”
Given that 1 in 13 Black-American children are born as carriers of sickle cell disease, and 1 in 365 are born with its symptoms, the new treatment may be a massive development for the Black community and offers a new treatment option for those living with the debilitating pain that comes with the disease.
“We know this drug can decrease the frequency of sickle cell pain crises in a significant and clinically meaningful way,” said Dr. Kenneth Ataga, Director of the Center for Sickle Cell Disease, University of Tennessee Health Science Center at Memphis, and Principal Investigator of the SUSTAIN trial. “The approval of [Adakveo] is an important advancement for people living with this very difficult condition.”
The decision to approve Adakveo was based on results of an extensive 52-week, randomized, placebo-controlled SUSTAIN trial, which showed that, “Adakveo significantly lowered the median annual rate of VOCs to 1.63 vs 2.98 compared to placebo (P=.010), which is equivalent to a 45% reduction.”
Approximately 100,000 people in the United States, most of whom are of African descent, live with sickle cell disease and the complications associated with the disease can often be life threatening. In addition to improving the quality of life for those affected, the treatment also has the potential to save Black families a significant amount of money on medical costs.
A study published in the journal Pediatric Blood and Cancer in 2009 found that in 2005 on average sickle cell disease cost an average of $11,702 for children covered by Medicaid insurance and a whopping $14,772 for those covered by employer-sponsored insurance.