Global Blood Therapeutics Inc said on Monday it plans to ask US regulators to greenlight the use of its sickle cell treatment, Oxbryta, in children aged 4 to 11, expanding on an earlier approval for patients 12 and over.
The South San Francisco, California-based company recently met with the US food and drug administration and plans to submit a formal application for expanded approval next year, it said in a statement.
The company is continuing to collect data supporting Oxbryta's safety and effectiveness in 4- to 11-year-olds but existing data is strong, chief executive Ted Love said in an interview.
"We've got a significant amount of data that really reinforces that the drug has a very similar profile for both safety and efficacy in this age group," Love said.
GBT's drug, known chemically as voxelotor, prevents red blood cells from sickling, a deformation that restricts oxygen flow in blood vessels, leading to severe pain and organ damage.
GBT will apply through the FDA's accelerated approval pathway, the company said in a statement.
The FDA approved Oxbryta in November to treat sickle cell disease in adults and children 12 years or older.
That came just a week after Novartis AG's Adakveo won US regulatory clearance to reduce the incidence of sickle cell-related pain crises, a common and debilitating symptom of the disease.
GBT's drug, known chemically as voxelotor, prevents red blood cells from sickling, a deformation that restricts oxygen flow in blood vessels, leading to severe pain and organ damage.
Sickle cell disease remains most prevalent in sub-Saharan African countries, where many of the estimated 300,000 children born annually with the condition may die before the age of 5. The disease is also common in India.