(Reuters) - Global Blood Therapeutics Inc said on Monday it plans to ask U.S. 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 U.S. 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 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 U.S. 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.
Reporting by Carl O’Donnell; Editing by Leslie Adler and Richard Chang
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