One of the few drugs for sickle cell disease is now available for children as young as 4 years old, a promising step towards expanding treatment options for a painful blood disease that has been underfunded in the past.
Global Blood Therapeutics, a San Francisco-based pharmaceutical company, received approval from the U.S. Food and Drug Administration in mid-December to lower the age limit for its daily oral medication, Oxbryta, from 12 years to 4 years. The drug is an option doctors may prescribe to help children manage the symptoms of sickle cell disease, for which there is no cure.
Sickle cell disease occurs when round red blood cells take on the C-shape of a farmer’s sickle and become hard and sticky, unable to carry enough oxygen to vital organs. In the US, the disease is most common – but not exclusive to – people of sub-Saharan African descent, with 1 in 365 black babies born with the disease.
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Until newborn screening became routine in the 1980s, few people born with sickle cell disease lived beyond childhood. Babies born with the disease today are increasingly living into adulthood, but little progress has been made in treating the disease since it was first recognized in the US in the early 1900s.
“One thing that’s been so exciting about the sickle cell journey is … to the extent that it’s really contributed to other areas of science, we’ve done little to develop therapies that are disease-modifying for people with sickle cell disease,” Kim Smith Whitley said. , the chief of research and development for GBT and the former director of the sickle cell program at Philadelphia Children’s Hospital.
Hydroxyurea, a cancer drug that has been used to treat sickle cell disease since the 1980s, is usually the first treatment doctors try on young patients, said Parul Rai, a pediatric hematologist at St. Jude Children’s Research Hospital.
When patients see no improvement with hydroxyurea, Rai and her colleagues try Oxbryta or Endari, which is approved for patients 5 years and older. Oxbryta helps restore the shape of red blood cells, reduce pain attacks and damage to other organs. The medication may be particularly helpful for children who are severely anemic (not enough red blood cells), or who would benefit from blood transfusions but cannot receive them.
Children who are at increased risk of stroke (the blood disease can affect how quickly blood flows to the brain) can be treated with monthly blood transfusions. But sometimes doctors can’t identify a good blood match or a family’s religion prevents them from getting transfusions, Rai said.
A 90-day supply of Oxbryta retails for about $13,840, while hydroxyurea costs an average of $79 for a 90-day supply, according to GoodRx, which tracks prescription drug prices. Both drugs are usually covered by insurance, which can drastically reduce their out-of-pocket costs.
Rai said there is no withheld demand for Oxbryta among her very young patients. But making the drug available to young children is a promising sign that more help is on the way for people with sickle cell disease.
“There just aren’t enough options,” Rai said. “The thing with sickle cell disease is that there are multiple pathways — it’s a waterfall of pain. Everything is interconnected… you can’t solve one problem and make it all better.”
As with any chronic health condition, the sooner patients can begin treatment to manage symptoms, the better: When managed properly, chronic health conditions are less likely to lead to other complications that can shorten a person’s lifespan.
Continuing research to develop new treatments for sickle cell disease and to determine whether existing drugs are safe for younger children could help children live healthier lives for longer.
“While a child with sickle cell disease has a higher chance of reaching adulthood today, one thing that is most sobering is that the transition from pediatric to adult-centered care is happening at a time when individuals with sickle cell disease tend to have more complications” , says Smith-Whitley. “The sooner we can initiate disease-modifying therapies, the more likely individuals are to transition healthily from pediatric to adult care.”